mycophenolic-acid and Dermatomyositis

Dermatomyositis (DM) is a rare inflammatory disorder affecting the muscle and skin. DM patients can present with spontaneous muscle hemorrhage, a potentially fatal complication. The best practice for management of hemorrhagic myositis in these patients remains unclear. Here we discuss the case of a patient who presented with progressive muscle weakness and intermittent rash that was diagnosed with dermatomyositis. During admission, she developed spontaneous hemorrhagic myositis of the right pectoralis major treated with surgical evacuation. She also developed a spontaneous left anterior thigh hematoma which was treated conservatively. She recovered and showed no evidence of recurrent bleeding at either location. We performed a literature review and identified ten cases of spontaneous hemorrhage in DM patients, with a 60% mortality rate among reported cases. Given the high mortality rate associated with spontaneous hemorrhage in DM patients, it is important for physicians to be aware of the diagnosis, workup, and management strategies.

Topics: Compression Bandages; Conservative Treatment; Dermatomyositis; Drainage; Enzyme Inhibitors; Female; Glucocorticoids; Hematoma; Hemorrhage; Hemostasis, Surgical; Humans; Hypotension; Immunoglobulins, Intravenous; Immunologic Factors; Methylprednisolone; Middle Aged; Mortality; Muscular Diseases; Mycophenolic Acid; Pectoralis Muscles; Prednisone; Quadriceps Muscle

Hypomyopathic dermatomyositis (HDM) is a rare form of dermatomyositis (DM). Interstitial lung disease (ILD) associated with clinically amyopathic DM (CADM-ILD) or hypomyopathic DM (HDM-ILD) is a rare condition with a more unfavorable prognosis than ILD associated with classic DM (CDM-ILD). There is no effective treatment for HDM-ILD. A 62-year-old woman with a 6-month history of chronic polyarthritis and myalgia presented skin lesions characteristic of DM (erythematous lesion on extensor surface of elbows, Gottron's papules, V-neck sign) with no clinical muscle impairment (global muscle strength: grade 5). Muscle enzymes (creatine kinase, lactic dehydrogenase, and aldolase) and electroneuromyography (ENMG) were normal. Computed tomography of the chest revealed ILD. Magnetic resonance imaging and muscle biopsy revealed subclinical muscle impairment. High doses of corticosteroids were used without success. As an alternative, 1500 mg/day of mycophenolate mofetil (MMF) was combined with low doses of prednisone, and the patient demonstrated a good clinical response after 3 months of this combination. Twenty-five months after initiating treatment, ILD remains in remission with the use of MMF and a low dose of prednisone. Therefore, MMF can be a good option for the treatment of HDM-ILD.

Topics: Anti-Inflammatory Agents; Dermatomyositis; Drug Therapy, Combination; Female; Humans; Lung Diseases, Interstitial; Middle Aged; Mycophenolic Acid; Prednisone; Treatment Outcome

In 2012, a European initiative called Single Hub and Access point for pediatric Rheumatology in Europe (SHARE) was launched to optimise and disseminate diagnostic and management regimens in Europe for children and young adults with rheumatic diseases. Juvenile dermatomyositis (JDM) is a rare disease within the group of paediatric rheumatic diseases (PRDs) and can lead to significant morbidity. Evidence-based guidelines are sparse and management is mostly based on physicians' experience. Consequently, treatment regimens differ throughout Europe.. To provide recommendations for diagnosis and treatment of JDM.. Recommendations were developed by an evidence-informed consensus process using the European League Against Rheumatism standard operating procedures. A committee was constituted, consisting of 19 experienced paediatric rheumatologists and 2 experts in paediatric exercise physiology and physical therapy, mainly from Europe. Recommendations derived from a validated systematic literature review were evaluated by an online survey and subsequently discussed at two consensus meetings using nominal group technique. Recommendations were accepted if >80% agreement was reached.. In total, 7 overarching principles, 33 recommendations on diagnosis and 19 recommendations on therapy were accepted with >80% agreement among experts. Topics covered include assessment of skin, muscle and major organ involvement and suggested treatment pathways.. The SHARE initiative aims to identify best practices for treatment of patients suffering from PRD. Within this remit, recommendations for the diagnosis and treatment of JDM have been formulated by an evidence-informed consensus process to produce a standard of care for patients with JDM throughout Europe.

Topics: Cyclosporine; Dermatomyositis; Europe; Evidence-Based Medicine; Exercise Therapy; Glucocorticoids; Humans; Immunosuppressive Agents; Methotrexate; Mycophenolic Acid; Patient Care Team; Practice Guidelines as Topic; Prednisolone; Rituximab; Societies, Medical; Sunscreening Agents

Dermatomyositis is a specific type of inflammatory myopathy with characteristic cutaneous findings. Patients may have skin disease without clinically apparent muscle disease, but this disorder is best thought of as a systemic process. Therefore, all patients with dermatomyositis skin lesions need appropriate evaluation for muscle disease, esophageal dysfunction, cardiopulmonary disease, and potential internal malignancy. There are many therapies that have been used for patients with dermatomyositis, but most are based upon case series or expert opinion rather than meta-analyses or randomized, placebo-controlled trials. Even those therapies that have been subjected to randomized, blinded, placebo-controlled trials include a mixture of patients with idiopathic inflammatory myopathy and do not utilize a validated assessment tool for measuring cutaneous disease responses. In this review, we discuss the therapies available as well as the internal associations with dermatomyositis.

Topics: Antibodies, Monoclonal, Murine-Derived; Dapsone; Dermatomyositis; Female; Humans; Immunoglobulins, Intravenous; Immunosuppressive Agents; Male; Methotrexate; Mycophenolic Acid; Prognosis; Risk Assessment; Rituximab; Stem Cell Transplantation; Treatment Outcome

To review the treatment advances of the inflammatory myopathies, a heterogeneous group of diseases that includes polymyositis, dermatomyositis, and inclusion body myositis.. There are few clinical trials in myositis, making it difficult to provide clear recommendations on the treatment of these rare disorders. The current management for IIM includes the initial use of corticosteroids followed by various conventional second-line treatments such as methotrexate and azathioprine. Although these drugs have not been tested in rigorous randomized controlled trials, general expert consensus confirms their use. Intravenous immunoglobulin is a reasonable short-term treatment with proven benefit in one controlled trial, although the evidence for other immunosuppressive therapies has been derived mainly from uncontrolled studies. Cyclosporine or tacrolimus have shown efficacy in myositis including those patients with interstitial lung disease (ILD), whereas mycophenolate mofetil is effective in both polymyositis and refractory dermatomyosits (including recalcitrant rash) and ILD. Uncontrolled studies for rituximab are encouraging but results from the largest randomized controlled trial in myositis failed to meet the primary endpoint. Anti-tumor necrosis factor (TNF) agents have shown mixed results in small, randomized clinical trials with infliximab demonstrating no benefit and etanercept leading to encouraging results warranting further study. Some newer novel therapies such as ACTH analogues and tocilizumab require additional investigation.. The balance of evidence suggests that traditional immunosuppressive and immunomodulatory drugs are certainly effective in polymyositis and dermatomyositis despite the lack of randomized controlled trials. Newer therapies are being studied but no major breakthroughs have been realized.

Topics: Azathioprine; Cyclosporine; Dermatomyositis; Drug Resistance; Drug Therapy, Combination; Glucocorticoids; Humans; Immunoglobulins, Intravenous; Immunomodulation; Immunosuppressive Agents; Methotrexate; Mycophenolic Acid; Polymyositis; Randomized Controlled Trials as Topic; Tacrolimus

Because polymyositis and dermatomyositis (PM/DM) are uncommon conditions, few randomized placebo controlled studies have been performed in these patients. The first line of therapy consists in high-dose oral prednisone, prescribed at 1mg/kg/day, then progressively tapered based on patients' clinical response. In patients who do not improve with corticosteroids alone, methotrexate is added, the therapeutic effect of which being observed within 8weeks. If PM/DM patients are refractory to corticosteroids and methotrexate, intravenous immunoglobulins can be added. In patients who fail to respond to this therapeutic strategy, it is crucial to make sure that the correct diagnosis has been made and we strongly recommend to perform a new muscle biopsy in order to exclude other myopathies. If the diagnosis of PM/DM is confirmed, a number of therapeutic agents may be proposed, including mycophenolate mofetil and rituximab. Importantly, TNF-α antagonists should not be considered in PM/DM patients, as these agents have been shown to favor exacerbation of interstitial lung disease and myositis and increase the risk of severe pyogenic and opportunistic infections in PM/DM patients.

Topics: Adrenal Cortex Hormones; Antibodies, Monoclonal, Murine-Derived; Dermatomyositis; Humans; Immunoglobulins, Intravenous; Immunologic Factors; Methotrexate; Mycophenolic Acid; Polymyositis; Randomized Controlled Trials as Topic; Rituximab; Treatment Outcome

Inflammatory myopathies are chronic, immune-mediated diseases characterized with progressive proximal muscle weakness. They encompass a variety of syndromes with protean manifestations. The aims of therapy are to increase muscle strength, prevent the development of contractures, and to manage the systemic manifestations of the disease. This is a complex treatment which requires routine and wide knowledge. The most important task is to recognize the disease and guide the patient to immunologic center. Although the first line of therapy continues to include corticosteroids, there are a multitude of agents available for treating patients with myositis. There are several different immunosuppressive agents which may be applied alone or in combination with each other, as well as an increasing number of novel and exciting biologic agents targeting molecules participating in the pathogenesis of inflammatory myopathy. Physiotherapy and rehabilitation in the remission period may significantly improve the functional outcome of patients with these disorders.

Topics: Adrenal Cortex Hormones; Algorithms; Antineoplastic Agents; Azathioprine; Biological Therapy; Cell Movement; Cyclophosphamide; Cyclosporine; Dermatomyositis; Exercise Therapy; Humans; Immunoglobulins, Intravenous; Immunosuppressive Agents; Methotrexate; Mycophenolic Acid; Paraneoplastic Syndromes; Physical Therapy Modalities; Polymyositis; T-Lymphocytes; Tacrolimus

Dermatomyositis (DM) is an idiopathic inflammatory myopathy. The mainstay of treatment for DM is oral corticosteroids. However, the dose and length of treatment is debated. Adding to the confusion, there have been no randomized controlled studies comparing the use of various corticosteroid doses and taper rates, and no controlled long-term studies assessing the hypothesis that, unlike systemic lupus erythematous, patients with DM can often achieve long-term remission off therapy. This literature review supports an approach that prednisone should be started at about 1 mg/kg/d, which is then tapered slowly based on the response. As patients respond differently to prednisone, additional therapies may be necessary. When to initiate these therapies requires clinical judgment. In addition, as we learn more about the pathophysiology of DM, newer medications that target specific mechanisms in the immune response may help us better treat the disease. Evidence-based data with long-term follow-up will allow for selection of the best treatment to maximize long-term remission, not simply short-term lowering of the systemic corticosteroid dose.

Topics: Adrenal Cortex Hormones; Alkylating Agents; Azathioprine; Cyclosporine; Dermatomyositis; Drug Administration Schedule; Exercise Therapy; Humans; Immunoglobulins, Intravenous; Methotrexate; Mycophenolic Acid; Plasma Exchange; Prognosis

Idiopathic inflammatory myositides are chronic diseases affecting predominantly the musculoskeletal system and involving high morbidity and mortality. Even when treated with glucocorticosteroids, patients often experience a progressive or relapsing course of the disease, requiring additional immunosuppressants, biologicals, or other therapeutic interventions. Randomized controlled trials on the treatment of primary myositides are still lacking, which means that the optimum therapeutic regimen has still not been defined. This article reviews the current position indicated by the data collected in standard and modern therapeutic options for the treatment of idiopathic inflammatory myositides.

Topics: Antibodies, Monoclonal; Antirheumatic Agents; Combined Modality Therapy; Dermatomyositis; Humans; Immunosuppressive Agents; Muscle Strength; Mycophenolic Acid; Polymyositis

Dermatomyositis is characterized by marked clinical heterogeneity. Many classification schemes are available, based on internal organ involvement, presence of circulating autoantibodies and additional symptoms characteristic for other connective tissue diseases. Viral infections and underlying malignancies are possible mechanisms to trigger this autoimmune disease. Intensive clinical investigations to detect such malignancies are therefore required. The prognosis of the individual patient depends on the involvement of internal organs and the characterization of circulating autoantibodies. There are patients with rapidly developing subsets of dermatomyositis and those with slowly progressive disease. Therefore treatment has to be adjusted for the disease severity. Sometimes high dosages of corticosteroids in combination with immunosuppressive agents or immunoglobulins are required.

Topics: Adult; Age Factors; Azathioprine; Biopsy; Child; Clinical Trials as Topic; Cyclophosphamide; Cyclosporine; Dermatologic Agents; Dermatomyositis; Disease Progression; Female; Humans; Immunoglobulins, Intravenous; Immunosuppressive Agents; Magnetic Resonance Imaging; Male; Methotrexate; Middle Aged; Muscles; Mycophenolic Acid; Paraneoplastic Syndromes; Plasmapheresis; Prognosis; Sex Factors; Time Factors

Self-resolving Epstein-Barr virus (EBV)-associated lymphomas have become more common with the use of immunosuppressive agents in both transplant patients and patients with connective tissue disorders. Immunosuppressive agents are often used for control of dermatomyositis, but their use has not been linked to subsequent malignancy. We present a 46-year-old woman with dermatomyositis, who developed an EBV-associated B-cell lymphoma of the brain while on oral methotrexate, mycophenolate mofetil and low-dose prednisone. The patient's lymphoma gradually resolved "spontaneously" upon discontinuation of the methotrexate and mycophenolate mofetil. The potential for EBV-associated B-cell lymphoma to self-resolve should be recognized by the clinician in order to prevent unnecessary and potentially toxic treatments including radiation therapy or multi-drug chemotherapy.

Topics: Biopsy; Brain Neoplasms; Burkitt Lymphoma; Dermatomyositis; Humans; Immunosuppressive Agents; Magnetic Resonance Imaging; Male; Methotrexate; Middle Aged; Mycophenolic Acid; Prednisone; Remission Induction; Withholding Treatment

It is well known that a certain percentage of patients with polymyositis and dermatomyositis (PM/DM) is corticosteroid resistant. Established and novel approaches to steroid-resistant PM/DM are discussed in this review. Methotrexate (MTX) is a first-line treatment in the case that steroid therapy fails. Azathioprine and cyclophosphamide also fall into this category. Cyclosporine, a specific inhibitor of calcineurin, has been reported almost as effective as MTX. Tacrolimus, also a calcineurin inhibitor, and mycophenolate mofetil could be additional alternatives for the treatment. Several clinical trials have demonstrated that high-dose intravenous immunoglobulin is promising. Recently favorable data have been published using intravenous high-dose pulse cyclophosphamide or cyclosporine for the poorly prognostic interstitial pneumonitis or pulmonary fibrosis accompanied with PM/DM.

Topics: Animals; Anti-Inflammatory Agents; Azathioprine; Cyclophosphamide; Cyclosporine; Dermatomyositis; Drug Resistance; Humans; Immunoglobulins, Intravenous; Immunosuppressive Agents; Lung Diseases, Interstitial; Methotrexate; Mycophenolic Acid; Polymyositis; Steroids; Tacrolimus

Topics: Adult; Aged; Creatine Kinase; Dermatomyositis; Drug Administration Schedule; Female; Humans; Immunoglobulins, Intravenous; Male; Mycophenolic Acid; Paraneoplastic Syndromes; Prednisolone; Prodrugs; Treatment Outcome

The objective of this study was to evaluate the efficacy and safety of MMF in juvenile idiopathic inflammatory myopathies (JIIMs).. Patients diagnosed with JIIM and treated with MMF enrolled in the Juvenile Dermatomyositis Research Group (JDRG) in the UK or followed at the Giannina Gaslini Institute in Genoa, Italy, were included. The following information was collected retrospectively at MMF initiation, at 3, 6 and 12 months after treatment start, and at last follow-up visit: clinical manifestations, laboratory data, physicians' subjective assessment of disease activity, standardized outcome measures of muscle strength/endurance, cutaneous disease activity, physical function, global disease activity, cumulative damage, and ongoing treatment.. Of the 29 patients included, 23 had juvenile DM and 6 had overlap myositis. During administration of MMF, improvement in measures of muscle strength, skin disease activity, and overall disease activity was seen, with an increase in the frequency of normal scores for Manual Muscle Test-8 from 50.0% to 83.3%, Childhood Myositis Activity Score from 53.5% to 88.9%, muscle component of DAS from 55.2% to 84.2%, skin component of DAS from 31.0% to 42.1%, visual analogue scale for skin disease activity from 25.0% to 47.4%, and visual analogue scale for overall disease activity from 7.1% to 42.1%. The number of patients with inactive disease increased from 10.3% at baseline to 68.5% at last follow-up. CS dose was significantly reduced, from 0.3 to 0.1 mg/kg/day. No relevant side effects were reported.. Our experience suggests that MMF is a valuable therapeutic option for the management of JIIM.

Topics: Child; Dermatomyositis; Humans; Mycophenolic Acid; Myositis; Retrospective Studies; Skin

Macrophage activation syndrome (MAS) is a severe and under-recognized complication of rheumatologic diseases. We describe a patient who presented with rapidly progressive, refractory MAS found to have anti-MDA5 antibody Juvenile Dermatomyositis (JDM) as her underlying rheumatologic diagnosis.. We describe a 14-year-old female who at the time of admission had a history of daily fevers for 6 weeks and an unintentional sixteen-pound weight loss. Review of systems was significant for cough, shortness of breath, chest pain, headaches, sore throat, muscle aches, rash, nausea, and loss of appetite. An extensive initial workup revealed findings consistent with an autoimmune process. While awaiting results of her workup she had clinical decompensation with multi-organ system involvement including pancytopenias, interstitial lung disease, hepatitis, cardiac involvement, gastrointestinal distension and pain, feeding intolerance, extensive mucocutaneous candidiasis, and neuropsychiatric decline. Due to her decompensation, significant interstitial lung disease, and likely underlying rheumatologic condition she was started on high dose pulse steroids and mycophenolate. An MRI was performed due to her transaminitis and shoulder pain revealing significant myositis. Intravenous immunoglobulin was then initiated. The myositis antibody panel sent early in her workup was significant for anti-MDA5 and anti-SSA-52 antibodies. Despite high dose pulse steroids, mycophenolate, and IVIG, her disease progressed requiring escalating therapies. Ultimately, she responded with resolution of her MAS as well as significant and steady improvement in her feeding intolerance, interstitial lung disease, cardiac dysfunction, myositis, arthritis, and cutaneous findings.. JDM in the pediatric patient is rare, as is MAS. In patients with complex rheumatologic conditions and lack of response to treatment, it is important to continually assess the patient's clinical status with MAS in mind, as this may change the treatment approach. Without proper recognition of this complication, patients can have a significant delay in diagnosis leading to life-threatening consequences.

Topics: Adolescent; Autoantibodies; Clinical Deterioration; Dermatomyositis; Dose-Response Relationship, Immunologic; Female; Glucocorticoids; Humans; Immunoglobulins, Intravenous; Immunologic Factors; Interferon-Induced Helicase, IFIH1; Macrophage Activation Syndrome; Magnetic Resonance Imaging; Multiple Organ Failure; Mycophenolic Acid; Pulse Therapy, Drug; Treatment Outcome

Topics: Dermatomyositis; Humans; Immunosuppressive Agents; Methotrexate; Mycophenolic Acid

We present two cases of Nodular Regenerative Hyperplasia (NRH) associated with Juvenile Dermatomyositis (JDM).. Case 1: A nine-year-old Caucasian male with refractory JDM and anti-NXP2 autoantibodies was diagnosed at age two. Over seven years, he developed arthritis, dysphagia, dysphonia, severe calcinosis, and colitis. Complications included recurrent cellulitis, infections, and hepatosplenomegaly. Multiple medications were chronically used, including prednisone, methotrexate, azathioprine, cyclophosphamide, mycophenolate mofetil, rituximab, tacrolimus, etanercept, abatacept, infliximab, and tocilizumab. Case 2: A 19-year-old Asian female with chronically active JDM and anti-MDA5 autoantibodies was diagnosed at age 15. Symptomatology included ulcerative skin lesions, Raynaud's phenomenon with digital ulcers, arthritis, interstitial lung disease with pulmonary hypertension, and calcinosis. Medications included chronic use of prednisone, methotrexate, abatacept, cyclophosphamide, mycophenolate mofetil, rituximab, tofacitinib, and sildenafil. In both patients, clinical symptomatology was not suggestive of liver disease or portal hypertension, but laboratory studies revealed elevated serum transaminases with progressive thrombocytopenia and no active liver-associated infections. The first patient's liver ultrasound showed coarse hepatic texture with mild echogenicity, splenomegaly, and portal hypertension. The second patient's liver ultrasound was normal, but elastography indicated increased stiffness. Liver biopsy confirmed NRH in both patients.. It is difficult to recognize NRH in JDM, as it often presents with elevated transaminases which may be mistaken for JDM muscle flare, corticosteroid-related fatty liver, or medication-related transaminitis. NRH has been associated with several medications used to treat JDM, including methotrexate, azathioprine, and cyclophosphamide, which should be discontinued if NRH develops. Providers should consider NRH in JDM patients with severe, refractory disease who have persistently elevated transaminases and persistent thrombocytopenia.

Topics: Abatacept; Adolescent; Arthritis; Autoantibodies; Azathioprine; Calcinosis; Child; Cyclophosphamide; Dermatomyositis; Female; Humans; Hyperplasia; Hypertension, Portal; Liver; Male; Methotrexate; Mycophenolic Acid; Prednisone; Rituximab; Splenomegaly; Thrombocytopenia; Transaminases; Young Adult

A 39-year-old woman admitted with multiple joint pain, hand rashes, and shortness of breath was diagnosed with anti-melanoma differentiation-associated gene 5 (MDA5) antibody-positive dermatomyositis (DM) with interstitial pneumonia (IP). Because of progressive dyspnoea and hypoxaemia, her IP was considered rapidly progressive interstitial lung disease. Initially, prednisolone 60 mg/day, cyclosporine A (CyA), and intravenous cyclophosphamide (IVCY) were initiated. A few days following the initiation of treatment, she experienced massive thunderclap headache, which was diagnosed as reversible cerebral vasospasm syndrome based on the findings of contraction in cerebral arteries with brain magnetic resonance imaging. Treatment with CyA and IVCY was discontinued, and diltiazem and mycophenolate mofetil (MMF) were initiated as an alternative immunosuppressant. Considering IVCY as the cause of Reversible cerebral vasospasm syndrome based on her clinical course, tacrolimus was commenced, which improved both DM and IP. DM patients who are anti-MDA5 antibody-positive are considered to have poor prognosis and require aggressive immunosuppressive treatments. In patients experiencing adverse events with standard IVCY, MMF with high-dose steroids and alternative calcineurin inhibitor should be considered.

Topics: Adult; Autoantibodies; Dermatomyositis; Disease Progression; Drug Therapy, Combination; Female; Humans; Immunosuppressive Agents; Interferon-Induced Helicase, IFIH1; Lung Diseases, Interstitial; Magnetic Resonance Imaging; Mycophenolic Acid; Tomography, X-Ray Computed; Vasospasm, Intracranial

Topics: Adolescent; Bayes Theorem; Child; Child, Preschool; Dermatomyositis; Female; Humans; Immunosuppressive Agents; Male; Mycophenolic Acid

Mesenteric panniculitis (MP), part of the spectrum of sclerosing mesenteritis, is an often asymptomatic disorder that is characterised by chronic inflammation of abdominal mesentery. We present a case of an 83-year-old woman who presented with proximal muscle weakness and erythematous, photosensitive rash of the face and upper torso and was subsequently diagnosed with dermatomyositis based on skin biopsy, electromyography and muscle biopsy. She had radiographic evidence of panniculitis on CT scan of the abdomen and pelvis for malignancy surveillance, which improved on serial CT scan 3 months after beginning treatment for her underlying dermatomyositis with prednisone and mycophenolate mofetil. Our case highlights that MP can be associated with underlying autoimmune disease. Connective tissue disease could be considered in the differential of MP when other etiologies such as surgery, trauma and malignancy are ruled out.

Topics: Aged, 80 and over; Anti-Inflammatory Agents; Dermatomyositis; Female; Humans; Mycophenolic Acid; Panniculitis, Peritoneal; Prednisone; Radiography; Tomography, X-Ray Computed; Treatment Outcome

Microsporidia are a group of obligate intracellular parasites that naturally infect domestic and wild animals. Human microsporidiosis is an increasingly recognized multisystem opportunistic infection. The clinical manifestations are diverse with diarrhea being the most common presenting symptom. We present a 52-year-old woman with a history of amyopathic dermatomyositis complicated by interstitial lung disease managed with mycophenolate mofetil and hydroxychloroquine who presented with a 7-month history of recurrent subcutaneous nodules as well as intermittent diarrhea and chronic sinusitis. A punch biopsy showed superficial and deep lymphocytic and granulomatous dermatitis with focal necrosis. Tissue stains for microorganisms revealed oval 1 to 3 μm spores within the necrotic areas in multiple tissue stains. Additional studies at the Centers for Disease Control and Prevention confirmed cutaneous microsporidiosis. This case is one of very few confirmed examples of cutaneous microsporidiosis reported in the literature.

Topics: Dermatomycoses; Dermatomyositis; Enzyme Inhibitors; Female; Humans; Hydroxychloroquine; Immunocompromised Host; Lung Diseases, Interstitial; Microsporidiosis; Middle Aged; Mycophenolic Acid

Immune-related adverse events (ir-AEs) are increasingly becoming a concern, as immune checkpoint inhibitors (ICIs) are used more frequently. Herein, we present a case of fulminant cytokine release syndrome (CRS) complicated by dermatomyositis after the combination therapy with ICIs.. A 70-year-old male developed dermatomyositis during the course of treatment with two ICIs, nivolumab and ipilimumab. He was treated by steroid pulse therapy, but the effect was limited. Afterwards, he had acute-onset high fever, hypotension, respiratory failure, impaired consciousness, renal failure, and coagulation abnormality at the same time. C reactive protein (CRP), creatinine kinase (CK), D-dimer, and ferritin levels were considerably elevated: CRP, 24 mg/dL; CK, 40,500 U/L; D-dimer, 290 μg/mL; ferritin, 329,000 ng/mL.. CRS induced by ICI combination therapy.. Given that high fever and elevated CRP level indicated potential sepsis, an antibiotic was used until the confirmation of negative blood cultures. All the simultaneous acute symptoms were supposed to be CRS. He was admitted to the intensive care unit (ICU), and temporary intubation and hemodialysis were needed. Immunosuppressive therapy was reinforced by mycophenolate mofetil together with steroid, and plasma exchange was performed for the elimination of abnormal proteins.. The patient's clinical symptoms and laboratory parameters gradually improved and he was discharged from the ICU in a month.. Fulminant CRS can be induced by ICI combination therapy. As the initial symptoms of CRS resemble sepsis, it is important to consider CRS as a differential diagnosis and to initiate immunosuppressive therapy early when needed. In steroid-resistant cases, early introduction of other immunosuppressive therapy and plasma exchange can be effective.

Topics: Aged; Antibiotics, Antineoplastic; Antineoplastic Agents, Immunological; Biomarkers; Combined Modality Therapy; Cytokine Release Syndrome; Dermatomyositis; Diagnosis, Differential; Humans; Immunologic Factors; Ipilimumab; Male; Mycophenolic Acid; Nivolumab; Plasma Exchange; Treatment Outcome

Antimelanoma differentiation-associated gene 5 (MDA-5) dermatomyositis is a subtype of dermatomyositis that is associated with rapidly progressive interstitial lung disease (RP-ILD), as well as with a variety of cutaneous manifestations. Patients with MDA-5 dermatomyositis tend to have a poor prognosis that is often attributed to the high rates of concurrent RP-ILD. Given the severity of disease, early diagnosis and aggressive management is pivotal. We present a case of a 40-year-old woman diagnosed with MDA-5 dermatomyositis who presented with weakness, painful cutaneous ulcerations and interstitial lung disease. She was treated with monthly intravenous Ig (IVIg), weight-based prednisone and mycophenolate mofetil (MMF). After approximately 2 years of treatment, her interstitial lung disease remains stable and she has had significant improvement in weakness and cutaneous ulcerations. Our case provides evidence for early and aggressive treatment of MDA-5 dermatomyositis with a combination of weight-based prednisone, MMF and IVIg.

Topics: Adrenal Cortex Hormones; Adult; Dermatomyositis; Female; Glucocorticoids; Humans; Immunoglobulins, Intravenous; Interferon-Induced Helicase, IFIH1; Lung Diseases, Interstitial; Mycophenolic Acid; Prednisone; Skin Ulcer

Topics: Antirheumatic Agents; Autoantibodies; Child, Preschool; Cyclophosphamide; Dermatomyositis; Diagnosis, Differential; Glucocorticoids; Humans; Male; Muscle Weakness; Mycophenolic Acid; Penis; Scrotum; Ulcer

Çakan M, Karadağ ŞG, Aktay Ayaz N. Complete and sustained resolution of calcinosis universalis in a juvenile dermatomyositis case with mycophenolate mofetil. Turk J Pediatr 2019; 61: 771-775. Juvenile dermatomyositis (JDM) is a rare, multisystemic, idiopathic vasculopathy mainly affecting the muscles and the skin. Gastrointestinal system, lungs, joints and heart may also be involved. Characteristic skin findings are heliotrope rash and Gottron papules but extensive skin involvement as large necrotic lesions are rarely reported. Calcinosis is one of the major issues in the long term. Delay in diagnosis, inadequate therapy at the initial phase, prolonged persistent disease activity are considered as major risk factors for the development of calcinosis. Treatment of calcinosis is also a major issue because no single treatment modality has been found to reproducibly stop or reverse calcification. A 5-year-old girl was admitted to our clinic with typical signs and symptoms of JDM. She was initially treated with high-dose corticosteroids, methotrexate and intravenous immunoglobulin (IVIG). Soon after, she developed necrotic ulcerative skin lesions and cyclosporine was added to her treatment regimen. By this treatment all muscle and skin manifestations were controlled but on the first year of follow-up she developed superficial calcification plaques on the upper extremities and calcinosis universalis like calcifications on the lower extremities. Calcifications did not respond to bisphosphonate (pamidronate) and IVIG treatment but mycophenolate mofetil resulted in rapid and sustained resolution of all calcification plaques.

Topics: Calcinosis; Child, Preschool; Dermatomyositis; Enzyme Inhibitors; Female; Humans; Mycophenolic Acid

Cutaneous disease represents a significant burden for patients with dermatomyositis. However, quantitative estimates of the probability of skin disease remission and clinical factors associated with skin outcomes are lacking.. To characterize cutaneous disease course in adult patients with dermatomyositis.. Prospective cohort study conducted at a dermatology clinic at a tertiary academic referral center. All adult patients with dermatomyositis (age >18 years) seen between May 15, 2007, and October 28, 2016, were eligible. Patients were included in the current analysis if they had a baseline Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) activity score of 12 or higher, and 2 or more CDASI scores separated by 3 months or more within their first 3 years of follow-up.. The percentage of patients who achieved clinical remission of their cutaneous disease as measured by the CDASI over a 3-year follow-up.. A total of 74 patients met our inclusion criteria (mean [SD] age at initial CDASI scoring, 54 [13] years; 58 women [78%]), and 28 (38%) achieved clinical remission during our 3-year follow-up period. Increased age (odds ratio [OR], 1.07; 95% CI, 1.02-1.12; P = .01), a dermatomyositis-associated malignancy (OR, 14.46; 95% CI, 2.18-96.07; P = .01), and treatment with mycophenolate mofetil (OR, 6.00; 95% CI, 1.66-21.78; P = .01) were significantly associated with clinical remission of skin disease in multivariable analysis. Patients with anti-melanoma differentiation-associated protein 5 antibodies had a significantly lower probability of meeting outcome criteria in our time-to-event analysis. Baseline cutaneous disease activity, disease duration at baseline, and disease duration before first systemic therapy were not significantly associated with clinical remission of skin disease.. Clinical remission was relatively uncommon in our population despite aggressive systemic therapy, and patients with anti-melanoma differentiation-associated protein 5 antibodies were even less likely to enter clinical remission during a 3-year follow-up period. Although mycophenolate mofetil compared favorably with other treatment options, our data provide evidence that a substantial population of patients with dermatomyositis have skin disease that is not adequately managed with standard-of-care therapies.

Topics: Academic Medical Centers; Adult; Analysis of Variance; Cohort Studies; Dermatologic Agents; Dermatomyositis; Female; Follow-Up Studies; Humans; Immunoglobulins, Intravenous; Kaplan-Meier Estimate; Logistic Models; Male; Methotrexate; Mycophenolic Acid; Prospective Studies; Risk Assessment; Severity of Illness Index; Statistics, Nonparametric; Time Factors; Treatment Outcome

Clinically amyopathic dermatomyositis (CADM) is a rare entity that presents with cutaneous manifestations of classic dermatomyositis but without muscle weakness or abnormal muscle enzymes. It is more common in young white and Asian females. A subset of patients with CADM has a specific antibody known as anti-MDA5. These patients have a more aggressive course with distinct cutaneous features, pulmonary involvement and early death. Here, we present the case of a 64-year-old Caucasian male with no significant medical history who was admitted with marked weight loss and a painful rash for 6 months. Patient had no muscle weakness and his rash was characteristic of classic dermatomyositis. Skin biopsy was suggestive of dermatomyositis but muscle enzymes were normal. His serum was positive for anti-MDA5 antibody. Extensive workup failed to detect any malignancy but he did show non-specific interstitial pneumonia. He was treated with prednisone and mycophenolate with good clinical response.

Topics: Anti-Inflammatory Agents; Autoantibodies; Dermatomyositis; Humans; Interferon-Induced Helicase, IFIH1; Lung Diseases, Interstitial; Male; Middle Aged; Mycophenolic Acid; Prednisone

We report on a 32-year-old male patient presenting with anti-MDA-5 and anti-Ro52 antibody positive hypomyopathic dermatomyositis (CADM) with clinically leading interstitial pulmonary involvement. Under several immunosuppressive treatment regimens including high-dose steroids, cyclophosphamide, rituximab, immunoglobulins, plasmapheresis, ciclosporin and mycophenolate mofetil, pulmonary involvement was refractory to progressive. Based on the detection of a clear-cut interferon signature by flow cytometric determination of SIGLEC-1 as an interferon-dependent marker, treatment with the Janus kinase inhibitor tofacitinib was initiated. This resulted in a response to treatment with a significant increase in physical performance, an ameliorated skin condition and computed tomographic (CT) morphologically improved interstitial lung disease with overall good tolerability.

Topics: Adult; Autoantibodies; Dermatomyositis; Humans; Immunosuppressive Agents; Janus Kinase Inhibitors; Lung Diseases, Interstitial; Male; Mycophenolic Acid

Currently, there are only few studies (mostly case reports or case series) on mycophenolate mofetil (MMF) in patients with systemic autoimmune myopathies (SAM). Therefore, the goal of the present study was to evaluate the safety and efficacy of MMF (monotherapy or coadjuvant drug) in a specific sample of patients with refractory SAM: dermatomyositis, polymyositis, anti-synthetase syndrome or clinically amyopathic dermatomyositis.. A case series including 20 consecutive adult patients with refractory SAM from 2010 to 2016 was conducted. After the introduction of MMF, associated or not with other drugs, the patients were followed for 6 consecutive months.. In 17 out of 20 patients MMF was introduced without any intolerance. The clinical symptoms evaluated in these patients were muscular, cutaneous and/or pulmonary activity. During the 6-month follow-up, 11 out of 17 patients had clinical and laboratory activities response with MMF, allowing significant tapering of the prednisone median dose (15 vs. 5 mg/day, P=0.005). On the other hand, in three out of 20 patients; MMF was discontinued in less than two months, because of gastrointestinal intolerance. There were no cases of serious infection or death.. MMF was relatively well-tolerated, safe and effective in patients with refractory SAM. Further studies are needed to confirm the data found.

Topics: Autoimmune Diseases; Dermatomyositis; Drug Therapy, Combination; Female; Glucocorticoids; Humans; Immunosuppressive Agents; Male; Middle Aged; Mycophenolic Acid; Myositis; Polymyositis; Prednisone; Retrospective Studies; Sex Factors; Statistics, Nonparametric

Topics: Autoantibodies; Dermatomyositis; Drug Therapy, Combination; Female; Hemoperfusion; Humans; Immunoglobulins, Intravenous; Immunosuppressive Agents; Interferon-Induced Helicase, IFIH1; Lung Diseases, Interstitial; Middle Aged; Mycophenolic Acid; Remission Induction; Rituximab; Tomography, X-Ray Computed; Treatment Outcome

Topics: Azathioprine; Dermatomyositis; Female; Humans; Immunoglobulins, Intravenous; Immunosuppressive Agents; Methotrexate; Middle Aged; Mycophenolic Acid; Piperidines; Pyrimidines; Pyrroles; Retreatment; Treatment Failure; Treatment Outcome

Topics: Administration, Oral; Dermatomyositis; Humans; Immunosuppressive Agents; Interferon-Induced Helicase, IFIH1; Lung Diseases, Interstitial; Male; Middle Aged; Mycophenolic Acid; Tomography, X-Ray Computed

Topics: Dermatomyositis; Female; Humans; Immunosuppressive Agents; Lung Diseases, Interstitial; Middle Aged; Mycophenolic Acid; Treatment Outcome

Topics: Biomarkers; Biopsy, Needle; DEAD-box RNA Helicases; Dermatomyositis; Drug Therapy, Combination; Electromyography; Enzyme-Linked Immunosorbent Assay; Follow-Up Studies; Gene Expression Regulation; Humans; Hydroxychloroquine; Immunohistochemistry; Interferon-Induced Helicase, IFIH1; Male; Methylprednisolone; Middle Aged; Mycophenolic Acid; Pityriasis Rubra Pilaris; Rare Diseases; Risk Assessment; Severity of Illness Index; Treatment Outcome

Topics: Aged; Dermatomyositis; Female; Humans; Immunosuppressive Agents; Mycophenolic Acid; Steroids

We reviewed our experience with immunosuppressive agents in patients with steroid-resistant Interstitial Lung Disease in the setting of Polymyositis/Dermatomyositis (PM/DM-ILD) to determine whether there were major differences in outcomes.. We identified all patients treated for PM/DM-ILD and assessed cyclophosphamide (CYC), azathioprine (AZA) and mycophenolate (MMF) when used as first-line steroid sparing therapy for effects on pulmonary function variables, dyspnea and tolerance at six and twelve months.. Among 46 patients meeting the inclusion criteria, 24 were treated with CYC, 13 with AZA and 9 with MMF. There were no baseline differences between the three treatment groups for any of the demographic or physiologic variables, dyspnea score, the presence of >30% fibrosis on CT, or the baseline steroid dose. At the six months assessment, the overall median change in FVC was 5.0% (25th, 75th percentile -3, 11.5%), corresponding to +.20 L (.09, 0.42 L) and the DLCO increased by 2.93% (-4, 9%), corresponding to 1 mm/ml/Hg (-.58, 2.3). The severity of dyspnea decreased substantially, prednisone dose could be reduced and no important difference in side effects was found in the whole group of patients. This effect was sustained after twelve months of therapy.. In patients with PM/DM-ILD related, treatment with CYC, AZA or MMF was associated with stabilization of pulmonary physiology, improved dyspnea, and a reduction of steroid dose.

Topics: Adult; Azathioprine; Cyclophosphamide; Dermatomyositis; Drug Administration Schedule; Drug Resistance; Drug Therapy, Combination; Dyspnea; Female; Glucocorticoids; Humans; Immunosuppressive Agents; Lung Diseases, Interstitial; Male; Middle Aged; Mycophenolic Acid; Polymyositis; Prednisone; Pulmonary Diffusing Capacity; Retrospective Studies; Treatment Outcome; Vital Capacity

Amyopathic dermatomyositis (ADM) causes a severe pulmonary disease in 50% of patients and no validated therapeutic option is available. Mycophenolate mofetil permitted to control interstitial lung disease and pneumomediastinum in a patient with ADM. This drug has recently been used to treat interstitial lung disease in ADM, but its effectiveness has not been previously reported in case of pneumomediastinum. Patients with ADM need an accurate pulmonary surveillance and in case of pulmonary complications mycophenolate mofetil could be a new therapeutic option.

Topics: Adult; Dermatomyositis; Enzyme Inhibitors; Humans; Lung Diseases, Interstitial; Male; Mediastinal Emphysema; Mycophenolic Acid

To date, no study has compared the clinical characteristics, malignancy associations, and treatment of dermatomyositis in predominantly Caucasian vs. Asian populations.. This prospective study was conducted to compare clinical characteristics of dermatomyositis, its relationship to malignancy, and treatment between two tertiary medical centers in the USA and Singapore. A total of 19 newly-diagnosed patients in the USA and 15 patients in Singapore were enrolled. Dermatomyositis or amyopathic dermatomyositis were diagnosed based on clinical assessment, skin and muscle biopsies, and muscle testing.. Ninety-five percent of patients in the USA group were of Caucasian descent, while 93% of patients in the Singapore group were of Chinese descent. Both groups were predominantly female. Pruritus was the most common initial symptom reported in both groups, while periungual erythema and Gottron's papules were the most common skin presentations. Heliotrope eruption was more common in the Singapore group, occurring in 80% of patients vs. 32% of patients in the USA group (P = 0.007). Three patients in the Singapore group developed a malignancy, with two of these patients having nasopharyngeal carcinoma. None of the USA patients developed malignancies in a follow- up period of 2-5 years. Immunosuppressive steroid sparing therapy with hydroxychloroquine was more frequently used in Singapore, while topical tacrolimus was more frequently used in the USA.. The clinical presentations of dermatomyositis vary among different ethnic populations. Chinese patients with dermatomyositis have a significant risk for nasopharyngeal carcinoma.

Topics: Adrenal Cortex Hormones; Adult; Aged; Antibodies, Monoclonal, Murine-Derived; Asian People; Bone Density Conservation Agents; Calcium Compounds; Carcinoma; Dermatomyositis; Dietary Supplements; Diphosphonates; Erythema; Female; Humans; Hydroxychloroquine; Immunosuppressive Agents; Male; Methotrexate; Middle Aged; Mycophenolic Acid; Nasopharyngeal Neoplasms; Oxides; Prospective Studies; Pruritus; Rituximab; Singapore; Tacrolimus; Tertiary Care Centers; United States; Vitamin D; White People

The objective of this study was to report the use of Mycophenolate Mofetil (MMF) in Juvenile Dermatomyositis (JDM). A retrospective chart review of children diagnosed with JDM having received MMF was performed. Response was evaluated 3 months after the onset of MMF by comparing muscle strength and steroid dosage before and after treatment. A good response was defined by global improvement concerning weakness and fatigability as evaluated subjectively by the physician along with a gain of at least 4 points on each of 2 muscle testings (Manual Muscle Testing, MMT and Childhood Myositis Assessment Score, CMAS) and/or a decrease of >15% of the corticosteroid dosage. Eight patients were identified. Except for one, all had received MMF secondary to an initial therapy of conventional immunosuppressants. Six patients showed good response by our predefined criteria. Changes of muscle testing scores ranged between +0 to +21 points (mean = +10.6) for the MMT and between +3 and +11 (mean = +7) for the CMAS. Corticosteroid tapering varied from 0 to 50%, with a mean of 18%. In most cases, follow-up was available for many months (up to 26); overall, we observed only one complication: a transient neutropenia in a patient concurrently receiving another immunosuppressant. This small series is the first published report on the use of MMF in JDM and suggests it is safe. Prospective larger studies are required to further elucidate the use of MMF in JDM.

Topics: Adolescent; Child; Child, Preschool; Dermatologic Agents; Dermatomyositis; Exercise Test; Female; Humans; Male; Muscle Strength; Muscle, Skeletal; Mycophenolic Acid; Recovery of Function; Retrospective Studies; Treatment Outcome

A 6-year-old girl, who had received corticosteroid and cyclosporine on the diagnosis of interstitial pneumonitis related to juvenile dermatomyositis, developed severe thrombocytopenia. Her thrombocytopenia was resistant to repeated intravenous immunoglobulin administration and methylprednisolone pulse therapy. After additional treatment with mycophenolate mofetil (MMF), instead of cyclosporine, the thrombocytopenia improved, facilitating a reduction in the dose of corticosteroid without exacerbation of the interstitial pneumonitis. We propose MMF as effective option in the treatment of immune thrombocytopenic purpura with autoimmune disease.

Topics: Child; Cyclosporine; Dermatomyositis; Drug Therapy, Combination; Female; Glucocorticoids; Humans; Immunoglobulins, Intravenous; Immunosuppressive Agents; Lung Diseases, Interstitial; Methylprednisolone; Mycophenolic Acid; Pulse Therapy, Drug; Purpura, Thrombocytopenic; Treatment Failure

Dermatomyositis is an inflammatory myopathy and commonly presents with progressive, symmetric proximal muscle weakness and cutaneous findings. Calcinosis is a severe manifestation that can be debilitating. The cutaneous manifestations of dermatomyositis may also develop in the absence of detectable muscle disease, and can persist after the successful treatment of myositis. The author reports a 30-year-old woman with biopsy-proven dermatomyositis who had failed previous trials of azathioprine and methotrexate. Her muscle weakness was controlled with mycophenolate mofetil and prednisone; however, she had recurrent attacks of painful calcinosis. The patient responded to intravenous immune globulin (IVIG) along with intravenous methylprednisone, followed by IVIG for 2 consecutive days each month. This regimen has been effective in preventing recurrence of her calcinosis. This case illustrates the cutaneous manifestation of dermatomyositis, which is often more refractory to treat as compared to the muscle involvement and require additional approaches such as IVIG.

Topics: Adult; Anti-Inflammatory Agents; Calcinosis; Dermatomyositis; Female; Humans; Immunoglobulins, Intravenous; Immunologic Factors; Immunosuppressive Agents; Methylprednisolone; Muscle Weakness; Mycophenolic Acid; Prednisone; Secondary Prevention

Juvenile dermatomyositis (JDM) and psoriasis are inflammatory disorders that share interferon-α induced responses and dysregulation of cytokines, including tumor necrosis factor alpha. Although 13% of patients with JDM have a family history of psoriasis there is little information concerning children with JDM and psoriasis.. We identified 3 children with both JDM and psoriasis. In 2 cases, psoriatic lesions occurred after the child's JDM symptoms had diminished following effective immunosuppressive therapy (high-dose intermittent intravenous methylprednisolone, methotrexate, and low dose oral corticosteroids). Patient 2, initially diagnosed as having psoriasis, was treated with prednisone and methotrexate but then developed classic JDM, which worsened following use of tumor necrosis factor alpha inhibitor and reduction of prednisone and methotrexate dosage. For each child, their history of JDM complicated the choice of therapy for psoriasis.. Two therapies commonly used to treat psoriasis-phototherapy and tumor necrosis factor-alpha antagonists-must be used with caution in patients with both JDM and psoriasis owing to their potential to exacerbate clinical manifestations of JDM. We discuss the implications affecting treatment of children with these dual diagnoses and consider the pathophysiology linking these 2 conditions.

Topics: Adolescent; Child; Child, Preschool; Cyclosporine; Dermatologic Agents; Dermatomyositis; Drug Therapy, Combination; Etanercept; Female; Humans; Immunoglobulin G; Methotrexate; Methylprednisolone; Mycophenolic Acid; Phototherapy; Prednisone; Psoriasis; Receptors, Tumor Necrosis Factor; Treatment Outcome; Tumor Necrosis Factor-alpha

Topics: Adrenal Cortex Hormones; Dermatomyositis; Drug Therapy, Combination; Female; Hand; Humans; Immunoglobulins, Intravenous; Immunosuppressive Agents; Middle Aged; Mycophenolic Acid; Necrosis; Skin; Treatment Outcome

We report a case of dermatomyositis associated with rheumatoid arthritis, Hashimoto thyroiditis, and diabetes mellitus responsive only to combination of rituximab with mycophenolate. A 42-year-old woman presented with proximal muscle weakness, myalgias, fever, night sweats, and shortness of breath. Creatinine kinase was 8155 IU/L, and muscle biopsy was diagnostic of dermatomyositis. She was started on glucocorticoids; her systemic symptoms improved, but her muscle weakness persisted. She was serially treated with intravenous immunoglobulin, azathioprine, and mycophenolate mofetil without improvement in her weakness. She responded dramatically to combination therapy with rituximab and mycophenolate, with improvement in strength and normalization of creatinine kinase. She has been well controlled on rituximab infusion every 6 months and maintenance mycophenolate mofetil.

Topics: Adult; Antibodies, Monoclonal, Murine-Derived; Antirheumatic Agents; Arthritis, Rheumatoid; Autoimmune Diseases; Dermatologic Agents; Dermatomyositis; Drug Therapy, Combination; Female; Hashimoto Disease; Humans; Mycophenolic Acid; Rituximab

To report our experience using mycophenolate mofetil as first-line treatment for dermatomyositis-associated interstitial lung disease.. We examined the medical records of all 16 dermatomyositis patients with interstitial lung disease seen in our outpatient university hospital dermatology clinic between May 26, 2006, and May 25, 2009. In this retrospective case series, we describe the clinical course of the 4 patients with definitive evidence of interstitial lung disease on radiologic imaging who were treated with mycophenolate mofetil and had pulmonary data available to document their outcome. All of the patients also received prednisone.. All 3 patients with at least 1 year of followup receiving mycophenolate mofetil experienced complete normalization of pulmonary function tests (including diffusing capacity for carbon monoxide) and resolution of dyspnea. They were also able to reduce their prednisone doses. The only patient with pre- and posttreatment chest computed tomography imaging had total resolution of her interstitial opacities. The patient with only 5 months of posttreatment followup experienced an improvement in diffusing capacity for carbon monoxide from 44% to 77% predicted, but no change in dyspnea.. These promising data indicate that mycophenolate mofetil may be a useful therapy for interstitial lung disease in patients with dermatomyositis, but larger studies are needed to more definitively evaluate the role of this medication in therapy.

Topics: Adult; Aged; Dermatomyositis; Female; Follow-Up Studies; Humans; Lung Diseases, Interstitial; Male; Middle Aged; Mycophenolic Acid; Retrospective Studies

To determine if mycophenolate mofetil (MMF) diminishes skin and muscle disease activity in children with juvenile dermatomyositis (DM), thereby permitting a decrease in corticosteroid dose.. A retrospective data review for 50 children with juvenile DM (mean ± SD age 12.2 ± 5.0 years) who had received MMF for 12 months identified the following characteristics: 38 (76%) were girls, 39 (78%) were white, 10 (20%) were Hispanic, and 1 (2%) was African American. The MMF dose and frequency, type of infection, white blood cell (WBC) count, corticosteroid dose, and the validated disease activity score (DAS) subscores for skin (DAS-S) and muscle (DAS-M) were obtained.. Twelve months after the start of MMF, the mean ± SD DAS-S decreased from 5.24 ± 0.29 to 3.72 ± 0.29 (P = 0.001), and the mean ± SD DAS-M decreased from 2.44 ± 0.39 to 1.17 ± 0.28 (P = 0.002). The mean ± SD prednisone dosage decreased from 0.39 ± 0.06 to 0.23 ± 0.02 mg/kg/day (P = 0.0001), with resumption of linear growth (P = 0.008). The WBC/lymphocyte count was unchanged over the 12 months on MMF. The infection rate was assessed in a subset of 26 children with juvenile DM who were observed for 12 months before the start of MMF and then compared with the ensuing 12 months of MMF therapy. There was no significant difference between the pretreatment period and the first 6 months of MMF therapy (P = 0.44), but the infection rate decreased in months 7-12 (P = 0.001).. MMF appears to be worthy of consideration as an additional therapeutic modality for treatment of children with juvenile DM. These data suggest that the use of MMF decreases skin and muscle disease activity and is steroid sparing. MMF appears to be well tolerated, but patients should be monitored for infection.

Topics: Adolescent; Age Factors; Child; Child, Preschool; Dermatomyositis; Female; Follow-Up Studies; Humans; Male; Mycophenolic Acid; Retrospective Studies; Young Adult

We report a second observation of autoimmune myelofibrosis associated with an inflammatory myositis in a 30-year-old female. The links between myelofibrosis and autoimmunity are discussed.

Topics: Adult; Anti-Inflammatory Agents; Autoimmune Diseases; Biopsy; Dermatologic Agents; Dermatomyositis; Drug Therapy, Combination; Female; Follow-Up Studies; Glucocorticoids; Humans; Mycophenolic Acid; Prednisone; Primary Myelofibrosis; Puerperal Disorders; Time Factors

To report the use of intravenous immunoglobulin (IVIg) and mycophenolate mofetil (MMF) in polymyositis (PM) and dermatomyositis (DM).. We performed an open study in PM and DM with active disease. Indications for treatment were: steroid-dependency, refractoriness to steroid and/or immunosuppressants, and life-threatening disease. IVIg was used at 2 g/kg in monthly cycles for six months and then each other month for other three cycles. MMF was slowly titrated to 30 mg/kg/day orally. Parameters employed to follow patients were the Medical Research Council (MRC) scale, the modified Rankin score, CK serum levels and daily prednisone dose.. Seven patients were studied (4PM, 3DM). All were females, with a mean age of 49 years. All of them achieved a complete remission and, at the last follow-up visit, significant differences in MRC score, modified Rankin score, CK levels, and the daily maintenance prednisone dose were documented. No relevant side effects were observed.. IVIg as add on treatment with MMF is effective in severe and refractory myositis, moreover as safe and steroid-sparing agent.

Topics: Aged; Creatine Kinase; Deglutition Disorders; Dermatomyositis; Disease Progression; Drug Resistance; Drug Therapy, Combination; Dyspnea; Female; Follow-Up Studies; Humans; Immunoglobulins, Intravenous; Immunosuppressive Agents; Middle Aged; Mycophenolic Acid; Polymyositis; Remission Induction

Lipodystrophy and metabolic abnormalities, primarily hypertriglyceridemia and insulin resistance, have been reported in juvenile dermatomyositis. We report a 55-year-old woman with adult dermatomyositis who developed lipodystrophy of the thighs, hypertriglyceridemia, and insulin resistance. Our case illustrates that lipodystrophy may occur in adult and juvenile dermatomyositis. Loss of subcutaneous tissue may be a cutaneous marker for metabolic abnormalities in both the adult and the juvenile forms of dermatomyositis.

Topics: Anti-Inflammatory Agents, Non-Steroidal; Dermatomyositis; Drug Therapy, Combination; Female; Glucocorticoids; Humans; Hypertriglyceridemia; Insulin Resistance; Lipodystrophy; Middle Aged; Mycophenolic Acid; Prednisone; Thigh

Dermatomyositis (DM) is a multisystem idiopathic inflammatory disorder that most commonly affects the muscles and skin. Systemic corticosteroids are the mainstay of therapy but are limited by their long-term adverse effects.. We sought to evaluate the effectiveness of oral mycophenolate mofetil in patients with cutaneous lesions of DM recalcitrant to other therapies through an open-label retrospective medical chart review of patients in a university-affiliated private practice setting. Twelve patients with DM who had skin lesions recalcitrant to traditional therapies or who developed toxic effects from traditional therapies began mycophenolate mofetil treatment at doses ranging from 500 mg to 1 g twice a day. Response was based on improvement in skin disease as judged clinically, an increase in strength, and/or an ability to decrease or discontinue concomitant therapies. Improvement was seen in 10 of the 12 patients, most within 4 to 8 weeks. Most patients tolerated mycophenolate mofetil treatment without problem; however, 1 patient developed a B-cell lymphoma of the central nervous system, and another developed abnormal levels of hepatic enzymes along with urinary symptoms. Resolution of these toxic reactions occurred with cessation of mycophenolate mofetil treatment in each patient.. Mycophenolate mofetil may be an effective corticosteroid-sparing therapy for the treatment of some patients with DM.

Topics: Adult; Dermatomyositis; Disease Progression; Drug Therapy, Combination; Female; Follow-Up Studies; Glucocorticoids; Humans; Immunosuppressive Agents; Male; Middle Aged; Mycophenolic Acid; Paraneoplastic Syndromes; Prodrugs; Retrospective Studies; Treatment Outcome

The authors report 10 patients with idiopathic dermatomyositis treated with mycophenolate mofetil in combination with corticosteroids. Successful steroid taper without disease relapse was achieved in six patients; however, in three patients, treatment was associated with opportunistic infections, leading to death in one patient. The disproportionately high rate of opportunistic infections in this group is considered.

Topics: Adult; Aged; Bronchopneumonia; Dermatomyositis; Drug Administration Schedule; Drug Therapy, Combination; Female; Humans; Immunosuppressive Agents; Male; Middle Aged; Mycophenolic Acid; Opportunistic Infections; Prednisone

Mycophenolate mofetil (MMF) is a new immunosuppressive agent currently being used for the prevention of renal allograft rejection. MMF is a specific inhibitor of lymphocytes and is well tolerated leading to its use in other autoimmune diseases. We have used MMF for the treatment of seven patients with inflammatory myopathy and are hereby reporting our results.. All of our patients were females (age 17-65 yr). They were symptomatic upon presentation and met classification criteria for idiopathic inflammatory myopathy. Inflammatory markers such as erythrocyte sedimentation rate and C-reactive protein as well as creatine kinase were significantly elevated in all the patients, indicating active disease. Corticosteroids were concomitantly being administered (20-60 mg/day of prednisone). Initial therapy with conventional immunosuppressives was either ineffective or had significant adverse effects leading to their discontinuation. MMF was started in doses of 500 mg twice a day and titrated up to 1 g twice a day.. Our patients have exhibited an impressive serological response to therapy with MMF and six patients had a marked improvement in their weakness. One patient had incomplete improvement in her weakness and has required additional therapies. MMF has been well tolerated during the treatment period (12-36 months).. A striking clinical and laboratory response of active myositis in six out of seven patients in this series illustrates that MMF can be effectively used in management of autoimmune inflammatory myopathy and may be a suitable alternative to the conventional immunosuppressive agents.

Topics: Adolescent; Adult; Aged; Autoimmune Diseases; Dermatomyositis; Female; Humans; Immunosuppressive Agents; Middle Aged; Muscle Weakness; Mycophenolic Acid; Myositis; Polymyositis; Treatment Outcome

Dermatomyositis (DM) and polymyositis (PM) are idiopathic inflammatory muscle diseases which remain a therapeutic challenge. The association between DM and malignancy is relatively well established while this relationship is weaker with PM. The clinical management and research for an occult malignancy as well as the follow-up of patients with DM or PM is a matter of debate. Herein we report a case of DM who, despite an extensive clinical, radiological and biological work-up developed an occult ovarian cancer 12 months after the initial diagnosis. This case report was used as support to review the actual expert recommendations for the search of an occult malignancy in presence of DM or PM.

Topics: Adenocarcinoma; Aged; Anti-Inflammatory Agents; Anti-Inflammatory Agents, Non-Steroidal; Azathioprine; Cyclosporins; Dermatologic Agents; Dermatomyositis; Diagnosis, Differential; Female; Glucocorticoids; Humans; Immunosuppressive Agents; Mycophenolic Acid; Ovarian Neoplasms; Polymyositis; Prednisone; Risk Factors; Time Factors

Four cases with classic skin manifestations and histologic evidence of dermatomyositis are presented. No occult malignancies were found. After conventional therapy with corticosteroids, hydroxychloroquine and/or methotrexate proved to be limited by side effects or an inadequate clinical response, a therapeutic trial of mycophenolate mofetil was instituted. This relatively new drug, which inhibits lymphocyte proliferation, was effective [with a mean duration of treatment of 13 months (range 6-20)] at controlling cutaneous disease activity, resulting in a decrease of the steroid dose.

Topics: Adult; Dermatomyositis; Exanthema; Female; Humans; Immunosuppressive Agents; Lymphocytes; Male; Middle Aged; Mycophenolic Acid; Treatment Outcome