Management of Myelofibrosis during Treatment with Ruxolitinib: A Real-World Perspective in Case of Resistance and/or Intolerance

Curr Oncol. 2022 Jul 15;29(7):4970-4980. doi: 10.3390/curroncol29070395.

Abstract

The development and approval of ruxolitinib, the first JAK1/2 inhibitor indicated to treat myelofibrosis, has improved patient outcomes, with higher spleen and symptoms responses, improved quality of life, and overall survival. Despite this, several unmet needs remain, including the absence of resistance criteria, suboptimal response, the timing of allogeneic transplant, and the management of patients in case of intolerance. Here, we report the results of the second survey led by the "MPN Lab" collaboration, which aimed to report physicians' perspectives on these topics. As in our first survey, physicians were selected throughout Italy, and we included those with extensive experience in treating myeloproliferative neoplasms and those with less experience representing clinical practice in the real world. The results presented here, summarized using descriptive analyses, highlight the need for a clear definition of response to ruxolitinib as well as recommendations to guide the management of ruxolitinib under specific conditions including anemia, thrombocytopenia, infections, and non-melanoma skin cancers.

Keywords: anemia; infection; intolerance; myelofibrosis; non-melanoma skin cancer; resistance; ruxolitinib; thrombocytopenia.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Anemia*
  • Humans
  • Nitriles
  • Primary Myelofibrosis* / diagnosis
  • Primary Myelofibrosis* / drug therapy
  • Pyrazoles / pharmacology
  • Pyrazoles / therapeutic use
  • Pyrimidines / therapeutic use
  • Quality of Life

Substances

  • Nitriles
  • Pyrazoles
  • Pyrimidines
  • ruxolitinib

Grants and funding

Editorial assistance was supported by an unconditional grant from Novartis Farma Italy.