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Jul 25, 2019

A virtual roundtable discussion with noted investigators Prof Claire Harrison and Dr John Mascarenhas for a review of recent innovations in the treatment of myeloproliferative neoplasms.

  • Biology of myeloproliferative neoplasms (MPN); mutational landscape and implications for prognosis and therapy (00:00)
  • Diagnosis and risk stratification of MPN (3:34)
  • Pathophysiology of MPN and therapeutic approach to patients with MPN (6:41)
  • Case (Prof Harrison): A man in his early 60s with symptomatic primary myelofibrosis (MF) and mutations in JAK2, ASXL1, EZH2 and TET2 receives ruxolitinib as first-line therapy (9:39)
  • Dosing and quality of life with ruxolitinib (11:58)
  • Case (Dr Mascarenhas): A man in his early 60s receives ruxolitinib for symptomatic primary MF and experiences a return of symptoms 3 years later (14:14)
  • Bone pain associated with MF; use of bone marrow biopsy to monitor disease progression (16:41)
  • Design, eligibility criteria and results of the Phase III COMFORT-I and COMFORT-II studies evaluating ruxolitinib for patients with MF (21:16)
  • A pooled analysis of overall survival in the COMFORT-I and COMFORT-II trials of ruxolitinib for the treatment of MF (23:53)
  • Predictors of overall survival benefit with ruxolitinib (26:01)
  • Biologic rationale for the survival benefit with ruxolitinib in MF (28:50)
  • Perspective on the use of ruxolitinib in patients with MF in the absence of symptoms (32:56)
  • Side-effect profile of ruxolitinib (36:36)
  • Management of cytopenias associated with ruxolitinib (40:33)
  • Potential correlation between JAK inhibitor therapy and second primary cancers (42:13)
  • Spectrum of activity and ongoing investigation of novel JAK inhibitors in MF (45:21)
  • Efficacy of fedratinib, pacritinib and momelotinib in patients with MF (48:08)
  • Activity and tolerability of fedratinib for MF (50:45)
  • Results of the single-arm, Phase II JAKARTA-2 study evaluating fedratinib in patients with MF previously treated with ruxolitinib (52:51)
  • Response to fedratinib in the JAKARTA-2 study; ongoing Phase III studies of fedratinib in the second-line setting for MF (55:49)
  • Case (Prof Harrison): A man in his late 60s with triple-negative primary MF experiences a good response to fedratinib as second-line therapy on the JAKARTA-2 trial (1:00:13)
  • Case (Dr Mascarenhas): A man in his early 70s with primary MF and cytopenias experiences an improvement in symptoms and reduction in spleen size after treatment with pacritinib on the PERSIST-2 study (1:03:23)
  • Potential role of fedratinib, pacritinib and momelotinib in the management of MF (1:05:11)
  • Ongoing investigation of erythroid maturation agents in patients with MF (1:09:09)
  • Perspective on the role of luspatercept and sotatercept in the management of MF (1:11:09)
  • Case (Prof Harrison): A man in his late 50s with severe pruritus associated with polycythemia vera (PV) experiences a dramatic relief of symptoms with ruxolitinib (1:13:11)
  • Efficacy of ruxolitinib in managing pruritus associated with PV (1:15:39)
  • Case (Dr Mascarenhas): A woman in her late 60s with essential thrombocythemia (ET) refuses cytoreductive therapy (1:18:24)
  • Effect of age and mutational status on the decision to treat ET (1:21:18)

CME information and select publications