ASH Clinical News March 2016 | Page 41

FEATURE David Fajgenbaum, MD, MBA, MSc David Fajgenbaum, MD, MBA, MSc, is an assistant professor of medicine at the University of Pennsylvania; associate director, patient impact for the UPenn Orphan Disease Center; and co-founder/executive director of the Castleman Disease Collaborative Network (CDCN). At age 25, during his third year of medical school, Dr. Fajgenbaum was diagnosed with idiopathic multicentric Castleman disease, a systemic form of Castleman disease that involves pro-inflammatory hypercytokinemia, reactive proliferation of lymphocytes, and life-threatening multiple organ system failure. His work researching the disease and connecting the global community of physicians, researchers, and patients has resulted in a major change in the way the disease is classified and has raised hopes that the disease’s etiology and pathogenesis will soon be elucidated. ASH Clinical News: Did you always intend to go into medicine? David Fajgenbaum, MD, MBA, MSc: I was always interested in health and medicine growing up. When I began college, I knew I wanted to become a physician, but that Nervous System Disorders 88 (22) 3 (1) 73 (19) 3 (1) Nervous System Disorders Peripheral neuropathiesb 2 (0) 167 (37) 23 (5) 103 (22) 5 (1) 113 (25) 10 (2) Psychiatric Disorders 63 (16) 6 (2) 50 (13) 8 (2) Respiratory, Thoracic, and Mediastinal Disorders Insomnia 1 (0) 46 (12) 0 Dyspneac 70 (18) 9 (2) 58 (15) 6 (2) 45 (12) 5 (1) 53 (14) 5 (1) Skin and Subcutaneous Tissue Disorders Dyspneac 123 (27) 23 (5) 66 (15) 8 (2) Cough 77 (17) 0 (0) 55 (12) 1 (0) 80 (17) 29 (6) 33 (7) 12 (3) Vascular Disorders Hypertensiond Vascular Disorders Embolic and thrombotic events, venousd 49 (13) 16 (4) 22 (6) 9 (2) Hypertensione 41 (11) 12 (3) 15 (4) 4 (1) KRd = Kyprolis, lenalidomide, and low-dose dexamethasone; Rd = lenalidomide and low-dose dexamethasone. a Pneumonia includes pneumonia and bronchopneumonia. b Peripheral neuropathies includes peripheral neuropathy, peripheral sensory neuropathy, and peripheral motor neuropathy. c Dyspnea includes dyspnea and dyspnea exertional. d Embolic and thrombotic events, venous include deep vein thrombosis, pulmonary embolism, thrombophlebitis superficial, thrombophlebitis, venous thrombosis limb, post thrombotic syndrome, venous thrombosis. e Hypertension includes hypertension, hypertensive crisis. Grade 3–4 Laboratory Abnormalities ( ≥10%) in Cycles 1–12 (20/27 mg/m2 Regimen in Combination with Lenalidomide and Dexamethasone) KRd (N = 392), n (%) Rd (N = 389), n (%) Decreased lymphocytes 182 (46) 119 (31) Decreased absolute neutrophil count 152 (39) 140 (36) Decreased phosphorus 122 (31) 106 (27) Decreased platelets 101 (26) 59 (15) Decreased total white blood cell count 97 (25) 71 (18) Decreased hemoglobin 58 (15) 68 (18) Decreased potassium 41 (11) 23 (6) KRd = Kyprolis, lenalidomide, and dexamethasone; Rd = lenalidomide and dexamethasone. Safety Experience with Kyprolis in Combination with Dexamethasone in Patients with Multiple Myeloma The safety of Kyprolis in combination with dexamethasone was evaluated in an open-label, randomized trial of patients with relapsed multiple myeloma. Patients received treatment for a median duration of 40 weeks in the Kyprolis/dexamethasone (Kd) arm and 27 weeks in the bortezomib/dexamethasone (Vd) arm. Deaths due to adverse reactions within 30 days of last study treatment occurred in 22/463 (5%) patients