Real-world outcomes of ruxolitinib treatment for polycythemia vera Journal Article
| Authors: | Coltoff, A.; Mesa, R.; Gotlib, J.; Shulman, J.; Rampal, R. K.; Siwoski, O.; Yacoub, A.; Moliterno, A.; Yang, A.; Braunstein, E.; Gerds, A. T.; Hobbs, G. S.; Winton, E. F.; Goel, S.; Wadleigh, M.; Tremblay, D.; Moshier, E.; Mascarenhas, J. |
| Article Title: | Real-world outcomes of ruxolitinib treatment for polycythemia vera |
| Abstract: | Introduction: Ruxolitinib is approved for the treatment of polycythemia vera (PV) with hydroxyurea resistance or intolerance. Approval was based on the phase III RESPONSE trial, which demonstrated efficacy in a highly selected patient population. Materials and Methods: To characterize the tolerability and outcomes of ruxolitinib outside of a clinical trial, we performed a multi-center retrospective analysis of patients with PV treated with ruxolitinib at 11 participating sites across the United States. Outcomes of interest included change in phlebotomy requirements after starting ruxolitinib and spleen response, as these were included in the primary composite outcome in the RESPONSE trial. Results: One hundred twenty-six patients met eligibility criteria, and the median duration of follow-up was 22.4 months (range, 0-63.0 months). At 32 weeks after starting ruxolitinib, the percentage of patients who received at least 1 phlebotomy was significantly decreased compared with before ruxolitinib (37% vs. 56%; relative risk [RR], 0.66; 95% confidence interval [CI], 0.52-0.84; P <.001). Phlebotomy requirements were similarly decreased in patients who had received at least 3 phlebotomies prior to ruxolitinib initiation (28% vs. 17%; RR, 1.65; 95% CI, 1.13-2.40; P <.01). Resolution of palpable splenomegaly was also documented (48% vs. 20%; RR, 2.45; 95% CI, 1.70-3.53; P <.0001). A total of 9.5% of patients discontinued ruxolitinib owing to treatment-emergent adverse events, and 81.7% of patients were receiving ruxolitinib at last known follow-up. Conclusion: These real-world results are similar to those reported from the RESPONSE trial, although additional follow-up is necessary to assess long-term outcomes and potential for late-onset toxicity. © 2020 Elsevier Inc. This is a retrospective study of patients with polycythemia vera treated with ruxolitinib. Data was collected from 11 participating centers across the United States. Ruxolitinib demonstrated efficacy in reducing phlebotomy requirements and palpable splenomegaly. This is the first real-world review of patients with polycythemia vera treated with ruxolitinib, and the results are similar to previously published clinical trials. © 2020 Elsevier Inc. |
| Keywords: | adult; controlled study; treatment response; aged; major clinical study; hydroxyurea; janus kinase 2; clinical trial; drug tolerability; diarrhea; drug efficacy; drug withdrawal; treatment duration; united states; comparative study; follow up; neoplasm; anemia; spleen; gastrointestinal symptom; tinnitus; clinical assessment; deep vein thrombosis; herpes zoster; retrospective study; risk factor; arthralgia; dizziness; depression; splenomegaly; urinary tract infection; response; transient ischemic attack; colitis; weakness; headache; memory disorder; cytopenia; adverse drug reaction; polycythemia vera; cerebrovascular accident; muscle cramp; cellulitis; allergic pneumonitis; myeloproliferative; clinical outcome; peginterferon alpha; soft tissue infection; anagrelide; phlebotomy; body weight disorder; breast tenderness; ruxolitinib; human; male; female; article; body weight gain; streptococcal pharyngitis |
| Journal Title: | Clinical Lymphoma, Myeloma and Leukemia |
| Volume: | 20 |
| Issue: | 10 |
| ISSN: | 2152-2650 |
| Publisher: | Elsevier Inc. |
| Date Published: | 2020-10-01 |
| Start Page: | 697 |
| End Page: | 703.e1 |
| Language: | English |
| DOI: | 10.1016/j.clml.2020.05.019 |
| PUBMED: | 32624445 |
| PROVIDER: | scopus |
| PMCID: | PMC8900057 |
| DOI/URL: | |
| Notes: | Article -- Jessica Schulman's name is misspelled as Shulman in the original publication -- Export Date: 2 November 2020 -- Source: Scopus |
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