Use of romiplostim for newly diagnosed immune thrombocytopenia in children

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Abstract

Immune thrombocytopenia (ITP) is a disease with a heterogeneous clinical manifestation. In the majority of children newly diagnosed ITP is a self-limited benign disorder, while chronic ITP develops rarely. The clinical onset of ITP can occur in very different ways: from nearly invisible skin hemorrhage to severe life-threatening bleeding. Conventional treatments promote a response in most patients, but in a small number of children thrombocytopenia is unresponsive. In this article, we describe our experience of the clinical use of romiplostim in children with severe unresponsive newly diagnosed ITP. The study was approved by the Independent Ethics Committee and the Scientific Council of the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology. The severity of bleeding decreased significantly after the start of romiplostim therapy in all cases. Durable complete (platelets > 100 × 109 /l) response was achieved in five out of six patients 4 to 8 weeks after starting therapy. Three children have remained in lasting remission for 1 to 3 years after the discontinuation of romiplostim. There were no adverse events associated with romiplostim.

About the authors

E. V. Suntsova

Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology Ministry of Healthcare of Russian Federation

Author for correspondence.
Email: elena.suncova@fccho-moscow.ru
ORCID iD: 0000-0001-8404-1800

MD, Department of hematopoiesis depressions, myeloid leukemias, rare and inherited diseases, 

117997, Moscow, Samory Mashela st., 1

Russian Federation

I. I. Chikvina

Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology Ministry of Healthcare of Russian Federation

ORCID iD: 0000-0002-5067-8288
Moscow Russian Federation

M. N. Sadovskaya

Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology Ministry of Healthcare of Russian Federation

ORCID iD: 0000-0002-7918-680X
Moscow Russian Federation

N. N. Kotskaya

Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology Ministry of Healthcare of Russian Federation

ORCID iD: 0000-0003-2787-6005
Moscow Russian Federation

L. A. Hachatryan

Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology Ministry of Healthcare of Russian Federation

ORCID iD: 0000-0001-7265-0414
Moscow Russian Federation

D. D. Baydildina

Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology Ministry of Healthcare of Russian Federation

ORCID iD: 0000-0001-7130-8596
Moscow Russian Federation

I. I. Kalinina

Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology Ministry of Healthcare of Russian Federation

ORCID iD: 0000-0002-0813-5626
Moscow Russian Federation

U. N. Petrova

Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology Ministry of Healthcare of Russian Federation

ORCID iD: 0000-0002-1258-8281
Moscow Russian Federation

A. V. Pshonkin

Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology Ministry of Healthcare of Russian Federation

ORCID iD: 0000-0002-2057-2036
Moscow Russian Federation

I. Ya. Lutfullin

Regional Children’s Hospital

ORCID iD: 0000-0002-0224-2746
Kazan Russian Federation

E. N. Lemazina

Regional Children’s Hospital

ORCID iD: 0000-0002-6569-6390
Kazan Russian Federation

I. V. Osipova

Regional Children’s Hospital

ORCID iD: 0000-0001-9833-5156
Kazan Russian Federation

A. V. Galeeva

Regional Children’s Hospital

Kazan Russian Federation

O. S. Naumova

Regional Children’s Hospital

ORCID iD: 0000-0002-6929-7338
Kazan Russian Federation

I. V. Fisyn

Scientific and Clinical Multidisciplinary Center for Help for Mothers and Children named after Z.I. Kruglaya

ORCID iD: 0000-0001-9050-7822
Oryol Russian Federation

G. V. Bikova

Regional Children’s Hospital

ORCID iD: 0000-0002-6552-4257
Stavropol Russian Federation

G. R. Kazaryan

Regional Children’s Hospital

ORCID iD: 0000-0002-1881-7444
Nizhnevartovsk Russian Federation

A. A. Maschan

Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology Ministry of Healthcare of Russian Federation

ORCID iD: 0000-0002-0016-6698
Moscow Russian Federation

G. A. Novichkova

Dmitriy Rogachev National Medical Research Center of Pediatric Hematology, Oncology, Immunology Ministry of Healthcare of Russian Federation

ORCID iD: 0000-0002-2322-5734
Moscow Russian Federation

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Copyright (c) 2020 Suntsova E.V., Chikvina I.I., Sadovskaya M.N., Kotskaya N.N., Hachatryan L.A., Baydildina D.D., Kalinina I.I., Petrova U.N., Pshonkin A.V., Lutfullin I.Y., Lemazina E.N., Osipova I.V., Galeeva A.V., Naumova O.S., Fisyn I.V., Bikova G.V., Kazaryan G.R., Maschan A.A., Novichkova G.A.

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