Rare Disease Report


The FDA has approved dasatinib (Sprycel) in combination with chemotherapy for the treatment of pediatric patients with newly diagnosed Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL).
The FDA has approved pembrolizumab for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC).
Mohamad Mohty, MD, PhD, discusses the investigational therapy cytotoxic T lymphocytes (CTLs) therapy for the treatment of Epstein-Barr virus associated post-transplant lymphoproliferative disorder (EBVPTLD). 
This rare leukemia form may be treated with ruxolitinib, which appears to be well-tolerated by patients.
Patients with myeloproliferative neoplasms (MPN) with essential thrombocythemia (ET) or polycythemia vera (PV) were found to benefit from hydroxyurea therapy. 
Patients with myeloid/lymphoid neoplasms enrolled in the phase 2 clinical trial demonstrated pemigatinib was generally well-tolerated.  
Nizar Bahlis, MD, highlights the phase 3 POLLUX trial evaluating daratumumab plus lenalidomide and dexamethasone for the treatment of patients with relapsed/refractory multiple myeloma. 
Lead investigator, John Leonard, MD, reviews the phase 3 AUGMENT trial investigating lenalidomide plus rituximab for the treatment of relapsed/refractory follicular lymphoma. 
Jorge Cortes, MD, highlights the phase 3 QuANTUM-R trial results and explains their clinical implications for patients with FLT3-ITD-mutated relapsed/refractory acute myeloid leukemia (AML). 
Jasmine Zain, MD, recaps the current and upcoming treatments in T-cell lymphoma and highlights the key clinical trials playing a role in the rare disease.
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