Clinical efficacy and safety of ruxolitinib in the management of myelofibrosis: A single institution experience in Taiwan

Yi Yang Chen, Cih En Huang, Kuan Der Lee, Chih Cheng Chen

Research output: Contribution to journalArticle

5 Citations (Scopus)

Abstract

OBJECTIVE: Myelofibrosis (MF) is a pathologic entity of myeloproliferative neoplasm (MPN) characterized by bone marrow fibrosis, extramedullary hematopoiesis, splenomegaly, and constitutional symptoms that severely affect the quality of life accompanied with the risk of leukemia development. Conventional treatment is usually ineffective and has limited impact on prolongation of survival. Dysregulated Janus kinase (JAK) signaling is common in MPN. In two randomized controlled trials, ruxolitinib, a potent pan-JAK inhibitor, has been shown to be highly effective in patients with intermediate- and high-risk MF.

METHOD: We retrospectively analyzed the therapeutic outcome of 10 MF patients treated with ruxolitinib in our institute. Basic clinical data, JAK2V617F mutational status and Myelofibrosis Symptoms Assessment Form (MF-SAF) to evaluate disease-related symptoms were recorded initially, and at every visit.

RESULT: Among these patients, only half of the patients harbored JAK2V617F mutation. After treatment with ruxolitinib, all patients had reduction of splenic size and reached nadir by week 24. Nine patients had body weight increment, and four of them had body weight increment more than 10%. Seven patients had their total symptom score reduced by more than 50% after therapy. The efficacy of ruxolitinib was irrelevant to JAK2V617F mutational status. Adverse events were mainly hematological and easily manageable.

DISCUSSION AND CONCLUSION: Ruxolitinib is both safe and efficacious in a cohort of Asian patients with MF. The efficacy of ruxolitinib is irrelevant to the mutational status of JAK2V617F.

Original languageEnglish
Pages (from-to)3-9
Number of pages7
JournalHematology (Amsterdam, Netherlands)
Volume21
Issue number1
DOIs
Publication statusPublished - Jan 1 2016

Fingerprint

Primary Myelofibrosis
Taiwan
Safety
Janus Kinases
Body Weight
Extramedullary Hematopoiesis
Symptom Assessment
INCB018424
Splenomegaly
Therapeutics
Neoplasms
Leukemia
Randomized Controlled Trials
Quality of Life
Mutation
Survival

Keywords

  • Asian population
  • INC424
  • JAK2 mutation
  • MF-SAF
  • Ruxolitinib
  • Splenomegaly

ASJC Scopus subject areas

  • Hematology

Cite this

Clinical efficacy and safety of ruxolitinib in the management of myelofibrosis : A single institution experience in Taiwan. / Chen, Yi Yang; Huang, Cih En; Lee, Kuan Der; Chen, Chih Cheng.

In: Hematology (Amsterdam, Netherlands), Vol. 21, No. 1, 01.01.2016, p. 3-9.

Research output: Contribution to journalArticle

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AB - OBJECTIVE: Myelofibrosis (MF) is a pathologic entity of myeloproliferative neoplasm (MPN) characterized by bone marrow fibrosis, extramedullary hematopoiesis, splenomegaly, and constitutional symptoms that severely affect the quality of life accompanied with the risk of leukemia development. Conventional treatment is usually ineffective and has limited impact on prolongation of survival. Dysregulated Janus kinase (JAK) signaling is common in MPN. In two randomized controlled trials, ruxolitinib, a potent pan-JAK inhibitor, has been shown to be highly effective in patients with intermediate- and high-risk MF.METHOD: We retrospectively analyzed the therapeutic outcome of 10 MF patients treated with ruxolitinib in our institute. Basic clinical data, JAK2V617F mutational status and Myelofibrosis Symptoms Assessment Form (MF-SAF) to evaluate disease-related symptoms were recorded initially, and at every visit.RESULT: Among these patients, only half of the patients harbored JAK2V617F mutation. After treatment with ruxolitinib, all patients had reduction of splenic size and reached nadir by week 24. Nine patients had body weight increment, and four of them had body weight increment more than 10%. Seven patients had their total symptom score reduced by more than 50% after therapy. The efficacy of ruxolitinib was irrelevant to JAK2V617F mutational status. Adverse events were mainly hematological and easily manageable.DISCUSSION AND CONCLUSION: Ruxolitinib is both safe and efficacious in a cohort of Asian patients with MF. The efficacy of ruxolitinib is irrelevant to the mutational status of JAK2V617F.

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