Developing strategies to reduce the duration of therapy for patients with myeloproliferative neoplasms.

Introduction: All current treatment strategies for myeloproliferative neoplasms (MPN) patients with the exception of allogeneic stem cell transplant (ASCT) are continuously administered. Treatment approaches that reduce the degree of minimal residual disease (MRD) might permit possible drug holidays or potential cures.

Area Covered: Authors discuss the presently available agents and those that are under clinical development that might induce a state of MRD and can be administered intermittently. Data extracted from a comprehensive search of peer review literature performed in Pubmed as well as information presented in scientific meetings.

Expert Opinion: Currently, the only potential curative treatment for MPN is ASCT. ASCT requires a period of intense treatment but ultimately allows the patient to enjoy a period independent of continued treatment. There is evidence that intermittent use of busulfan or prolonged use of IFN-α can induce hematological remissions that are sustained for prolonged periods of time, allowing for drug holidays. The experimental drug Imetelstat is a promising drug that has been reported to prolong survival in very high-risk myelofibrosis patients after a limited period of time of administration. New experimental drugs and drug combinations that target the malignant clone and/or microenvironmental abnormalities have the potential to eliminate MRD, which might allow for drug holidays and reduction in the duration of therapy.