Patrizia Mondello1, Cristian Di Mirto2, Salvatore Cuzzocrea3, Carmela Arrigo2, Michael Mian4, Vincenzo Pitini2. 1. Department of Medicine, Division of Hematology and Medical Oncology, Weill Cornell Medical College, New York, NY; Department of Human Pathology, University of Messina, Messina, Italy; Department of Biological and Environmental Sciences, University of Messina, Messina, Italy. Electronic address: pam2068@med.cornell.edu. 2. Department of Human Pathology, University of Messina, Messina, Italy. 3. Department of Biological and Environmental Sciences, University of Messina, Messina, Italy. 4. Internal Medicine V, Hematology and Oncology, Medical University of Innsbruck, Innsbruck, Austria; Department of Hematology and CBMT, Ospedale di Bolzano, Bolzano, Italy.
Abstract
BACKGROUND: Despite the important progress in the research of myeloproliferative neoplasms (MPN), treatment options are still limited. Currently, a cytoreductive approach is the backbone treatment, with hydroxyurea (HU) being the most important agent. However, this drug is not always well-tolerated and has been questionably linked to a potential leukemogenic effect. A valid alternative is interferon alfa (IFN-α), but it is reserved for selected patients owing to the more frequent side effects and the lack of final results from the studies directly comparing IFN-α with HU, which is why we provided the results of the so far largest real-life analysis. PATIENTS AND METHODS: From 2000 to 2016, 63 patients with Philadelphia-negative MPN prospectively received either HU or IFN-α. RESULTS: During a median follow-up period of 121 months (range, 88-168 months), 97% of the patients treated with IFN-α achieved a hematologic response (60% complete, 37% partial) compared with 78% in the HU group (56% complete, 20% partial; P < .01). Molecular responses were limited to patients treated with IFN-α. IFN-α was well-tolerated with no secondary malignancy, whereas HU was associated with more toxic events and cases of leukemic transformation. A significantly longer progression-free survival (5.0 vs. 3.1 years; P < .001) and overall survival (7.8 vs. 5.8 years; P = .006) were observed in the IFN-α group compared with the HU cohort. CONCLUSION: Our data support IFN-α as a more valid therapeutic option owing to its more profound hematologic responses, durable molecular remissions, long-term disease control, and reduced risk of leukemic transformation with a favorable toxicity profile.
BACKGROUND: Despite the important progress in the research of myeloproliferative neoplasms (MPN), treatment options are still limited. Currently, a cytoreductive approach is the backbone treatment, with hydroxyurea (HU) being the most important agent. However, this drug is not always well-tolerated and has been questionably linked to a potential leukemogenic effect. A valid alternative is interferon alfa (IFN-α), but it is reserved for selected patients owing to the more frequent side effects and the lack of final results from the studies directly comparing IFN-α with HU, which is why we provided the results of the so far largest real-life analysis. PATIENTS AND METHODS: From 2000 to 2016, 63 patients with Philadelphia-negative MPN prospectively received either HU or IFN-α. RESULTS: During a median follow-up period of 121 months (range, 88-168 months), 97% of the patients treated with IFN-α achieved a hematologic response (60% complete, 37% partial) compared with 78% in the HU group (56% complete, 20% partial; P < .01). Molecular responses were limited to patients treated with IFN-α. IFN-α was well-tolerated with no secondary malignancy, whereas HU was associated with more toxic events and cases of leukemic transformation. A significantly longer progression-free survival (5.0 vs. 3.1 years; P < .001) and overall survival (7.8 vs. 5.8 years; P = .006) were observed in the IFN-α group compared with the HU cohort. CONCLUSION: Our data support IFN-α as a more valid therapeutic option owing to its more profound hematologic responses, durable molecular remissions, long-term disease control, and reduced risk of leukemic transformation with a favorable toxicity profile.