mycophenolic-acid and Hemophilia-A

Topics: Child; China; Hemophilia A; Humans; Immunosuppressive Agents; Kidney Transplantation; Mycophenolic Acid

Topics: Hemophilia A; Humans; Immunosuppressive Agents; Mycophenolic Acid; Omalizumab; Pemphigoid, Bullous

Topics: Anti-Inflammatory Agents; Blood Coagulation Factors; Factor VIIa; Hemophilia A; Humans; Immunoglobulins, Intravenous; Immunosuppressive Agents; Infant; Male; Methylprednisolone; Mutation; Mycophenolic Acid; Recombinant Proteins; Vasculitis; Wiskott-Aldrich Syndrome; Wiskott-Aldrich Syndrome Protein

Topics: Aged; Aged, 80 and over; Female; Hemophilia A; Humans; Male; Middle Aged; Mycophenolic Acid; Treatment Outcome

Acquired haemophilia A (AHA) is a bleeding disorder that results from autoantibodies against factor VIII (FVIII). A 70-year-old man with a history of interstitial lung disease presented with spontaneous bleeding into his thigh. He had undetectable FVIII levels and a high-titre FVIII inhibitor (>2000ââ'¬â€°Bethesda units/mL) and was diagnosed with AHA. He had several relapses, required multiple haemostatic and immunosuppressive treatments but eventually achieved a stable remission after 2ââ'¬â€°years of therapy.Our patient matches the typical elderly male demographic of AHA. His relapsing course with remarkably high and persistent inhibitor titre highlights the need for close monitoring and aggressive upfront treatment. Whereas cyclophosphamide and steroids are often used first line in AHA, rituximab has also shown efficacy in refractory patients with high inhibitor levels. The FVIII and inhibitor concentration on presentation have been associated with treatment response and may be used as prognostic factors to tailor immunosuppressive regimens.

Topics: Aged; Antibiotics, Antineoplastic; Flank Pain; Follow-Up Studies; Hemophilia A; Hemorrhage; Humans; Lung Diseases, Interstitial; Male; Mycophenolic Acid; Remission Induction; Secondary Prevention; Thigh; Time Factors; Treatment Outcome

Here, we report about a boy (age: 18 years) who developed an acquired factor VIII inhibitor at the age of 9 years. He presented with bleeding in his right ankle, multiple haematomas and a high-titer factor VIII type II inhibitor (400 BU).. He received treatment with MMF (CellCept®), dexamethasone-immunoglobulin pulses, and rituximab together with high dose FVIII (Hannover protocol). His inhibitor titer decreased rapidly, and half-life and recovery normalized. Inhibitor titres increased after reduction of the factor VIII dose, and increased further after MMF was stopped. A second treatment course with MMF again resulted in reduction of the titre, improvement in half life and recovery, and no more bleedings. Inhibitor reappeared with MMF dose reduction, again accompanied by severe bleeding. Additional rituximab stopped the bleedings, and treatment with MMF has been continued since.. Although the laboratory parameters showed no complete remission, severe bleedings and expensive factor replacement could be avoided by long-term treatment with MMF.

Topics: Adolescent; Blood Coagulation Factor Inhibitors; Factor VIII; Hemophilia A; Humans; Immunosuppressive Agents; Longitudinal Studies; Male; Mycophenolic Acid; Treatment Outcome

Topics: Adult; Anti-Inflammatory Agents; Blood Coagulation Factor Inhibitors; Cyclophosphamide; Enzyme Inhibitors; Female; Hemophilia A; Humans; Immunosuppressive Agents; Mycophenolic Acid; Partial Thromboplastin Time; Prednisolone; Remission Induction; Treatment Outcome

Induction of factor VIII (FVIII) inhibitors sometimes occurs in patients with hemophilia due to frequent supplementation of FVIII. The inhibitor is rarely detected in non-hemophilic patients; however, an association has been described in patients with chronic inflammatory diseases, such as autoimmune diseases (e.g. SLE and rheumatoid arthritis), malignant tumors and drug allergies, and also to pregnant or aged individuals without underlying disease. We report on an 82-year-old patient who was transferred to our hospital after the diagnosis of acquired FVIII inhibitor. On admission the laboratory results showed no detectable FVIII activity (0%, normal range 70-100%), prolongation of coagulation time (APTT 102.4 s), and severe anemia 7.8 g/dL (normal range 12-16 g/dL). On physical examination multiple subcutaneous hematomas were detected and further bleeding in his left pectoralis muscle was observed. Despite extensive investigation no underlying disease was detected. Thirty-six courses of plasma exchange with 360 units of fresh frozen plasma replacement daily were conducted. High dose steroids and mycophenolate mofetil (MMF) were given throughout the course, and cyclophosphamide was administered once. Thirty-four units of erythrocytes were applied during this time. After 36 courses of plasma exchange in combination with high dose steroids, FVIII activity and coagulation time normalized and bleeding could be stopped. The patient was discharged in good health 48 days after admission. Hence, we present a case of severe idiopathic FVIII inhibitor-positive hemophilia successfully treated with the combination of plasma exchange, corticosteroids, cyclophosphamide and MMF.

Topics: Adrenal Cortex Hormones; Aged, 80 and over; Combined Modality Therapy; Cyclophosphamide; Erythrocyte Transfusion; Factor VIII; Follow-Up Studies; Hemophilia A; Humans; Infusions, Intravenous; Male; Mycophenolic Acid; Plasma Exchange; Risk Assessment; Severity of Illness Index; Treatment Outcome

Topics: Adolescent; Autoantibodies; Drug Therapy, Combination; Factor VIII; Female; Hemophilia A; Humans; Immunosuppressive Agents; Mycophenolic Acid; Prednisone; Treatment Outcome