1. MDS-191: Long-Term Efficacy and Safety of Luspatercept in Lower-Risk Myelodysplastic Syndromes (MDS): Phase 2 PACE-MDS Study
- Author
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Carolyn Barron, Xiaosha Zhang, Ulrich Germing, Karin Mayer, Markus P. Radsak, Joseph G. Reynolds, Joerg Chromik, Uwe Platzbecker, Philipp Kiewe, Abderrahmane Laadem, Kenneth M. Attie, Katharina Götze, Thomas Wolff, and Aristoteles Giagounidis
- Subjects
myalgia ,Cancer Research ,medicine.medical_specialty ,Erythema ,business.industry ,Myelodysplastic syndromes ,Peripheral edema ,Phases of clinical research ,Hematology ,medicine.disease ,Lower risk ,Clinical trial ,Oncology ,hemic and lymphatic diseases ,Internal medicine ,medicine ,medicine.symptom ,Bone pain ,business - Abstract
Background: Luspatercept, a first-in-class erythroid maturation agent, has been investigated in patients with LR-MDS and ring sideroblasts (RS) (MEDALIST; Fenaux and Platzbecker NEJM 2020) and in an ongoing Phase 3 trial regardless of RS status (COMMANDS, NCT03682536 ). The previously reported Phase 2 trial of luspatercept (Platzbecker Lanc Onc 2017) includes subtypes of LR-MDS with and without RS, regardless of prior ESA exposure, and various EPO levels. Aims: Evaluate the long-term safety and efficacy of luspatercept in LR-MDS. Methods: Patients were IPSS low/int-1, age ≥ 18 years, Hgb NCT01749514 ; NCT02268383 ). Results: As of 13July2019, 115 patients were enrolled, of whom 108 were treated with ≥0.75 mg/kg. 44/108 (41%) were non-RS; 47/108 (44%) had prior ESA; 45/108 (42%) had baseline transfusion burden of ≥4U RBC/8 weeks. Median duration of treatment (n=108) was 10.4 (0.7-57.3) months. IWG HI-E RR was 54% (58/108) overall; 36% (16/44) in non-RS, 68% (42/62) in RS+ (2 patients had unknown RS status); 53% (25/47) prior ESA, 54% (33/61) in ESA-naive; 54% (34/63) in 2 patients) were fatigue, headache, hypertension, arthralgia, bone pain, diarrhea, injection site erythema, myalgia, and peripheral edema. Conclusions: Consistent with previous reports, data from the long-term Phase 2 study of luspatercept in LR-MDS continues to show efficacy in a variety of subtypes and safety similar to previous and ongoing studies.
- Published
- 2020
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