Defining ruxolitinib failure and transition to next-line therapy for patients with myelofibrosis: a modified Delphi panel consensus study

Author:

Mascarenhas John1ORCID,Nguyen Hiep2,Saunders Ashley2,Oliver Louisa3,Tomkinson Hannah3,Perry Richard3ORCID,McBride Ali2

Affiliation:

1. Icahn School of Medicine at Mount Sinai, New York, NY 10029, USA

2. Bristol Myers Squibb, Princeton, NJ 08540, USA

3. Adelphi Values PROVE, Manchester, SK10 5JB, UK

Abstract

Aim: To define ruxolitinib failure and develop parameters to guide transition to next-line therapy for patients with myelofibrosis. Methods: A modified Delphi panel with 14 hematologists-oncologists. Survey concepts included defining primary refractory status, loss of response, disease progression, intolerance and transition to next-line therapy. Results: Ruxolitinib failure may be defined as no improvement in symptoms or spleen size, progressive disease or ruxolitinib intolerance, following a maximally tolerated dose for ≥3 months. Loss of spleen response 1 month after initial response may prompt discontinuation. Lack of evidence to inform transition to next-line therapy was noted; tapering ruxolitinib should be considered according to ruxolitinib dose and patient characteristics. Conclusion: Expert consensus was provided on defining ruxolitinib failure and transition to next-line therapy as summarized in this position paper, which may support considerations in the development of future clinical practice guidelines.

Funder

Bristol-Myers Squibb

Publisher

Future Medicine Ltd

Subject

Cancer Research,Oncology,General Medicine

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