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Setting Appropriate Goals for the Next Generation of Clinical Trials in Myelofibrosis

  • Myeloproliferative Disorders (C Harrison, Section Editor)
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Abstract

New targeted therapies administered in phase II and phase III studies have produced substantial improvements in outcomes of myelofibrosis (MF). However, strong documentation that the new agents modify the natural history of the disease is lacking, and a number of therapeutic indications of new drugs remain unaddressed. Overall survival (OS) improvement is the major goal of next-generation clinical trials in MF. This may be attained if an adequate population of patients and an unambiguous design of the trial will be selected. Another goal is preventing disease progression in early MF: this requires a controlled clinical trial with an accessible endpoint and a clinically relevant definition of disease progression. Improvement in the documentation of responsiveness of patient-reported outcomes (PROs) will allow to use them as a critical endpoint of new trials. Finally, exploiting the clinical utility of biomarkers should become a major goal of future clinical experimentation in MF.

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Acknowledgments

GB was supported by a grant from Associazione Italiana per la Ricerca sul Cancro (AIRC, Milano) “Special Program Molecular Clinical Oncology 5x1000” to AGIMM (AIRC-Gruppo Italiano Malattie Mieloproliferative).

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Giovanni Barosi declares no potential conflicts of interest.

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This article does not contain any studies with human or animal subjects performed by any of the authors.

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Correspondence to Giovanni Barosi.

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This article is part of the Topical Collection on Myeloproliferative Disorders

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Barosi, G. Setting Appropriate Goals for the Next Generation of Clinical Trials in Myelofibrosis. Curr Hematol Malig Rep 10, 362–369 (2015). https://doi.org/10.1007/s11899-015-0281-2

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