The efficacy and safety of romiplostim in the treatment of thrombocytopenia in pediatric patients with Wiskott–Aldrich syndrome: the results of a retrospective study

A. L. Khoreva; Хорева А. Л.; I. N. Abramova; Абрамова И. Н.; E. V. Deripapa; Дерипапа Е. В.; Yu. A. Rodina; Родина Ю. А.; A. A. Roppelt; Роппельт А. А.; V. I. Burlakov; Бурлаков В. И.; D. E. Pershin; Першин Д. Е.; S. S. Larin; Ларин С. С.; E. V. Raykina; Райкина Е. В.; T. V. Varlamova; Варламова Т. В.; A. M. Kieva; Киева А. М.; K. A. Voronin; Воронин К. А.; A. A. Maschan; Масчан А. А.; G. A. Novichkova; Новичкова Г. А.; A. Yu. Shcherbina; Щербина А. Ю.

doi:10.24287/1726-1708-2020-19-4suppl-39-49

The efficacy and safety of romiplostim in the treatment of thrombocytopenia in pediatric patients with Wiskott–Aldrich syndrome: the results of a retrospective study

Authors: Khoreva A.L.¹, Abramova I.N.¹, Deripapa E.V.¹, Rodina Y.A.¹, Roppelt A.A.¹, Burlakov V.I.¹, Pershin D.E.¹, Larin S.S.¹, Raykina E.V.¹, Varlamova T.V.¹, Kieva A.M.¹, Voronin K.A.¹, Maschan A.A.¹, Novichkova G.A.¹, Shcherbina A.Y.¹
Affiliations:
1. Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology of Ministry of Healthcare of the Russian Federation
Issue: Vol 19, No 4 (2020): supplement
Pages: 39-49
Section: MATERIALS OF THE 3rd WORKING MEETING OF ALLERGISTS-IMMUNOLOGISTS
Submitted: 08.01.2021
Accepted: 08.01.2021
Published: 08.01.2021
URL: https://hemoncim.com/jour/article/view/445
DOI: https://doi.org/10.24287/1726-1708-2020-19-4suppl-39-49
ID: 445

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Reduction of haemorrhage and improving the quality of life for patients with Wiskott–Aldrich syndrome (WAS) before hematopoietic stem cell transplantation (HSCT) are the most important goals. We aimed to investigate the efficacy and safety of thrombopoietin receptor agonist (TPO-RA) in children with WAS. This study is supported by the Independent Ethics Committee and approved by the Academic Council of the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology. 67 patients with genetically confirmed WAS treated with subcutaneous romiplostim at the dose of 9 µg/kg weekly between 2012 and 2019 in Dmitry Rogachev National Center of Pediatric Hematology, Oncology, and Immunology were enrolled. The median treatment duration was 8 months (range, 1‒12). Overall, 22 patients (33%) achieved complete response and 18 (27%) partial. There were no apparent severe adverse effects associated with romiplostim administration. The only notable and possibly not related to the treatment was arterial thrombosis in one patient who had ongoing systemic vasculitis and a history of aortic aneurysm formation. The results of our study demonstrated that use of romiplostim was safe and effective in managing of thrombocytopenia and bleeding in most WAS patients.

Keywords

Wiskott–Aldrich syndrome, romiplostim, thrombocytopenia, haemorrhagic syndrome

MD, PhD, Professor, Deputy Director, Institute of Hematology, Immunology and Cellular Technologies; Chief of Immunology Department

1 Samory Mashela St., Moscow 117997, Russia

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The efficacy and safety of romiplostim in the treatment of thrombocytopenia in pediatric patients with Wiskott–Aldrich syndrome: the results of a retrospective study

Full Text

Abstract

Keywords

About the authors

References

Supplementary files