melphalan and Hypertension--Pulmonary

POEMS syndrome is a clonal plasma cell disease characterized by polyneuropathy, organomegaly, endocrinopathy, M protein, and skin changes. Significant advances have been made in the diagnosis and treatment of POEMS syndrome over the last decade. Herein, the diagnostic criteria and characteristic features are reviewed, focusing the role of characteristic features in early diagnoses. Autologous peripheral blood stem cell transplantation has become the first-line treatment for younger patients with normal organ function. Autologous transplantation has resulted in a high response rate and durable remission. Moreover, transplantation-related morbidity and mortality has been significantly reduced over the past 5 years. Induction therapy before transplantation may improve the harvest of stem cells and decrease transplantation-related morbidity. Melphalan and dexamethasone is an effective and well-tolerated treatment for older patients or those with organ dysfunction. Novel agents may also offer benefits to patients with a poor performance status or renal dysfunction, and transform transplantation eligibility.

Topics: Antibodies, Monoclonal, Humanized; Bevacizumab; Boronic Acids; Bortezomib; Castleman Disease; Chromosome Aberrations; Combined Modality Therapy; Dexamethasone; Disease Management; Drug Therapy, Combination; Genes, Immunoglobulin; Humans; Hypertension, Pulmonary; Lenalidomide; Melphalan; Osteosclerosis; Peripheral Blood Stem Cell Transplantation; Plasma Cells; POEMS Syndrome; Prognosis; Pyrazines; Severity of Illness Index; Symptom Assessment; Thalidomide; Transplantation, Autologous; Vascular Endothelial Growth Factor A

The optimal autologous stem cell rescue (HDC-SCR) regimen for children with high-risk neuroblastoma (HR-NBL) is not defined. Carboplatin/etoposide/melphalan (CEM) is the current US standard; however, European data suggest busulfan/melphalan (Bu/Mel) may have less toxicity. Published data regarding toxicities associated with CEM and Bu/Mel are limited. We conducted a single-institution retrospective cohort study of children with HR-NBL who received CEM or Bu/Mel preparative regimens. Toxicity data were analyzed using χ(2) or Fisher's exact, Wilcoxon two-sample or log-rank tests. Sinusoidal obstruction syndrome (SOS) was observed in 7/44 CEM (15.9%) and 5/21 (24%) Bu/Mel patients (P=0.50). Median time to SOS was longer following Bu/Mel than CEM (20 versus 9 days, P=0.02). Pulmonary hypertension (PHTN) was observed in ~20% of children after Bu/Mel and none after CEM (P=0.01). CEM patients had more nephrotoxicity (P=0.001), packed red blood cell (P=0.02) and platelet transfusions (P=0.008), and days on maximum pain support (P=0.0007). Time to engraftment, length of stay, documented infection rates and HDC-SCR-related mortality were similar. Nephrotoxicity and resource utilization associated with cytopenias and mucositis were greater after CEM. Pulmonary toxicities were more severe after Bu/Mel, and increased vigilance for PHTN may be warranted, particularly in children with hypoxemia out of proportion to respiratory distress.

Topics: Adolescent; Antineoplastic Combined Chemotherapy Protocols; Busulfan; Carboplatin; Child; Child, Preschool; Cohort Studies; Etoposide; Female; Hepatic Veno-Occlusive Disease; Humans; Hypertension, Pulmonary; Infant; Kidney Diseases; Male; Melphalan; Mucositis; Myeloablative Agonists; Neuroblastoma; Pancytopenia; Retrospective Studies; Stem Cell Transplantation; Transplantation Conditioning; Young Adult

We experienced the case of a 67-year-old man with refractory heart failure. He presented with dyspnea and progressive pitting edema of the lower limbs. Diuretics were insufficient to improve his symptoms. Cardiac catheterization demonstrated pulmonary hypertension. Additional examinations confirmed polyneuropathy, organomegaly, endocrinopathy and monoclonal gammopathy. The plasma vascular endothelial growth factor (VEGF) level was 1,340 pg/mL. The patient was diagnosed with Crow-Fukase (POEMS) syndrome. Echocardiography detected left ventricular hypertrophy and diastolic dysfunction. Polysomnography demonstrated severe sleep-disordered breathing. We herein describe a case of pulmonary hypertension with Crow-Fukase syndrome accompanied by left ventricular diastolic dysfunction and sleep-disordered breathing.

Topics: Aged; Dexamethasone; Diuretics; Drug Resistance; Edema; Furosemide; Heart Failure; Humans; Hypertension, Pulmonary; Hypertrophy, Left Ventricular; Male; Melphalan; POEMS Syndrome; Polysomnography; Prednisolone; Pulmonary Wedge Pressure; Sleep Apnea Syndromes; Thalidomide; Ultrasonography; Vascular Endothelial Growth Factor A; Ventricular Dysfunction, Left

Topics: Adult; Antineoplastic Agents, Alkylating; Antineoplastic Combined Chemotherapy Protocols; Biopsy; Bone Marrow; Bone Marrow Examination; Dexamethasone; Diagnosis, Differential; Doxorubicin; Echocardiography, Transesophageal; Follow-Up Studies; Humans; Hypertension, Pulmonary; Male; Melphalan; Multiple Myeloma; Peripheral Blood Stem Cell Transplantation; POEMS Syndrome; Radiography, Thoracic; Time Factors; Treatment Outcome; Tricuspid Valve Insufficiency; Ultrasonography, Doppler, Color; Vincristine