학술논문
Romiplostim for Treatment of Children and Young Adults With Severe Aplastic Anemia and Myelodysplastic Syndrome.
Document Type
Academic Journal
Author
Sharathkumar A; Stead Family Department of Pediatrics, Carver College of Medicine.; Holden Comprehensive Cancer Center.; Carr J; Institute for Clinical and Translational Science.; Claassen D; Stead Family Department of Pediatrics, Carver College of Medicine.; Syrbu S; Department of Pathology, University of Iowa, Iowa City, IA.; Bhagavathi S; Department of Pathology, University of Iowa, Iowa City, IA.; Al-Huniti A; Department of Pediatrics, Mayo Clinic, Hematology, Rochester, MN.; Modi A; Department of Pediatrics, University of Arkansas for Medical Sciences, Little Rock, AR.; Bates M; Holden Comprehensive Cancer Center.; Department of Health and Human Physiology.; Department of Internal Medicine, Carver College of Medicine, University of Iowa, Iowa City, IA.; Mott SL; Holden Comprehensive Cancer Center.
Source
Publisher: Lippincott Williams & Wilkins Country of Publication: United States NLM ID: 9505928 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1536-3678 (Electronic) Linking ISSN: 10774114 NLM ISO Abbreviation: J Pediatr Hematol Oncol Subsets: MEDLINE
Subject
Language
English
Abstract
Thrombopoietin receptor agonists (TPO-RAs) induce trilineage hematopoiesis under conditions with acquired hematopoietic failure. We evaluated safety, tolerability, and preliminary efficacy of a TPO-RA, romiplostim (Nplate), with or without standard-of-care immunosuppressive therapy (±IST) for children (ages <21 y) with newly diagnosed and relapsed/refractory severe aplastic anemia (SAA) and myelodysplastic syndrome (MDS). Data were collected from an observational study and a single arm interventional pilot study. The safety outcome was treatment-related adverse events (AEs). Efficacy was evaluated by complete hematopoietic response (CHR) at week 24. Romiplostim was commenced at 5 µg/kg/week, with dose escalation of 2.5 µg/kg/week (maximum, 20 µg/kg/dose) based on platelet response. Romiplostim was continued until CHR was observed. Ten subjects (SAA, 9 [IST, 4; without IST, 5]; MDS, 1) completed the study (median age: 9.2 y). Median romiplostim dose was 10 µg/kg/week (range: 5 to 17.5 µg/kg/week). The cumulative incidence of CHR was 70.4% (95% CI, 20.2%-92.6%). Among 21 AEs (Grade 1 to 3), 3 were attributed to romiplostim. At a median posttherapy follow-up of 10.9 months (range: 0.7 to 77.5), no clonal evolution, bone marrow fibrosis or mortality was reported. This proof-of-concept study provides data about short-term safety, tolerability, and preliminary efficacy of romiplostim (±IST) for treatment of pediatric SAA/MDS.
Competing Interests: A.S. received funding from Amgen, Inc, to conduct this pilot study under investigator sponsored study category. Funding agency has no role in study design, data analyses and publication of the manuscript. M.B. is founder and chief executive officer of LSF Medical Solutions whose work does not overlap topically with this work. The other authors declare no conflict of interest.
(Copyright © 2024 Wolters Kluwer Health, Inc. All rights reserved.)
Competing Interests: A.S. received funding from Amgen, Inc, to conduct this pilot study under investigator sponsored study category. Funding agency has no role in study design, data analyses and publication of the manuscript. M.B. is founder and chief executive officer of LSF Medical Solutions whose work does not overlap topically with this work. The other authors declare no conflict of interest.
(Copyright © 2024 Wolters Kluwer Health, Inc. All rights reserved.)