Affiliation: Mount Sinai School of Medicine
- Mascarenhas J. Rationale for combination therapy in myelofibrosis. Best Pract Res Clin Haematol. 2014;27:197-208 pubmed publisher..This manuscript will address the proposed goals of combination therapy approach and review the state of the art in combination experimental therapy for MF. ..
- Mascarenhas J, Talpaz M, Gupta V, Foltz L, Savona M, Paquette R, et al. Primary analysis of a phase II open-label trial of INCB039110, a selective JAK1 inhibitor, in patients with myelofibrosis. Haematologica. 2017;102:327-335 pubmed publisher..Non-hematologic adverse events were largely grade 1 or 2; the most common was fatigue. Treatment with INCB039110 resulted in clinically meaningful symptom relief, modest spleen volume reduction, and limited myelosuppression. ..
- Mascarenhas J, Sandy L, Lu M, Yoon J, Petersen B, Zhang D, et al. A phase II study of panobinostat in patients with primary myelofibrosis (PMF) and post-polycythemia vera/essential thrombocythemia myelofibrosis (post-PV/ET MF). Leuk Res. 2017;53:13-19 pubmed publisher..The optimal dosing of panobinostat for the treatment of MF remains somewhat ill-defined but appears to be most effective and better tolerated when administered at lower doses over a prolonged duration of therapy. ..
- Mascarenhas J. A Concise Update on Risk Factors, Therapy, and Outcome of Leukemic Transformation of Myeloproliferative Neoplasms. Clin Lymphoma Myeloma Leuk. 2016;16 Suppl:S124-9 pubmed publisher..Current JAK2 inhibitor-based combination therapy approaches are supported by preclinical investigation and are currently being tested in multicenter clinical trials. ..
- Lee S, Salehi I, Chary M, Schiano T, Mascarenhas J. "Irony" of managing refractory anemia with transfusional support in hemophagocytic lymphohistiocytosis. Transfus Apher Sci. 2016;55:105-8 pubmed publisher..After receiving sixteen units of packed RBCs, liver iron concentration rose to 6.7?mg/g dry weight, which is highly unusual in other diseases requiring transfusional support. ..
- Zahr A, Salama M, Carreau N, Tremblay D, Verstovsek S, Mesa R, et al. Bone marrow fibrosis in myelofibrosis: pathogenesis, prognosis and targeted strategies. Haematologica. 2016;101:660-71 pubmed publisher..We discuss the rationale of various anti-fibrogenic treatment strategies targeting the clonal hematopoietic stem/progenitor cell, aberrant signaling pathways, fibrogenic cytokines, and the tumor microenvironment. ..
- Choi D, Tremblay D, Iancu Rubin C, Mascarenhas J. Programmed cell death-1 pathway inhibition in myeloid malignancies: implications for myeloproliferative neoplasms. Ann Hematol. 2017;96:919-927 pubmed publisher..The current understanding of immune dysfunction in MPNs and analogous MyMs offers a compelling rationale to study PD-1/PD-L1 inhibition in patients as a novel treatment option. ..
- Tremblay D, Marcellino B, Mascarenhas J. Pharmacotherapy of Myelofibrosis. Drugs. 2017;77:1549-1563 pubmed publisher..This review highlights the current landscape of MF pharmacotherapy and explores therapeutic advances underway. ..
- Keyzner A, Han S, Shapiro S, Moshier E, Schorr E, Petersen B, et al. Outcome of Allogeneic Hematopoietic Stem Cell Transplantation for Patients with Chronic and Advanced Phase Myelofibrosis. Biol Blood Marrow Transplant. 2016;22:2180-2186 pubmed publisher..Baseline splenomegaly did not affect transplantation outcomes. Given the particularly poor outcomes seen in the unrelated donor cohort here and elsewhere, a formal exploration of alternative hematopoietic stem cell sources is warranted. ..
- Carreau N, Tremblay D, Savona M, Kremyanskaya M, Mascarenhas J. Ironing out the details of iron overload in myelofibrosis: Lessons from myelodysplastic syndromes. Blood Rev. 2016;30:349-56 pubmed publisher..This manuscript reviews known and potential implications of IO in MF and highlights the need for prospective clinical investigations of ICT with consideration in the setting of JAK2 inhibitor therapy. ..