Severe Thrombocytopenia Due to Bone Marrow Failure in Children With Dyskeratosis Congenita Does Not Respond to Eltrombopag Treatment: Case Series

被引：1

作者：

Pramanik-Jonsson, Lotta ^{[1
,2
,6
]}

Borssen, Magnus ^{[4
,5
]}

Vonlanthen, Sofie ^{[3
]}

Nilsson, Frans ^{[4
,5
]}

Sundin, Mikael ^{[1
,2
]}

机构：

[1] Karolinska Inst, Dept Clin Sci Intervent & Technol, Div Pediat, Stockholm, Sweden

[2] Astrid Lindgren Childrens Hosp, Sect Pediat Hematol Immunol & HCT, Solna, Sweden

[3] Karolinska Univ Hosp, Karolinska Inst, Dept Clin Immunol & Transfus Med, SE-17176 Stockholm, Sweden

[4] Umea Univ, Dept Med Biosci, Umea, Sweden

[5] Univ Hosp Umea, Child & Adolescent Med Ctr, Sect Pediat Hematol & Oncol, Umea, Sweden

[6] Karolinska Univ Hosp, Astrid Lindgren Childrens Hosp, Sect Pediat Hematol Immunol & HCT, SE-14186 Stockholm, Sweden

来源：

JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY | 2024年 / 46卷 / 01期

关键词：

eltrombopag; dyskeratosis congenita; thrombocytopenia; bone marrow failure; CHRONIC IMMUNE THROMBOCYTOPENIA; MULTICENTER;

D O I：

10.1097/MPH.0000000000002775

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

Dyskeratosis congenita is a rare inherited disease with classic cutaneous symptoms, sometimes accompanied with more severe extracutaneous manifestations such as bone marrow failure, which can be lethal. Eltrombopag is an orally available thrombopoietin receptor agonist in clinical use for increasing platelet levels in patients with immune thrombocytopenia and aplastic anemia. Here, 3 pediatric patients with dyskeratosis congenita are presented with varying disease severity, in which off-label eltrombopag treatment had no clinical effect on bone marrow failure. This, in addition to the negative results in a previous case report, supports the preclusion of eltrombopag use in dyskeratosis congenita.

引用

页码：57 / 62

页数：6

共 2 条

[1] Up-front Treatment With Romiplostim in Children With Acquired Bone Marrow Failure: A Single Institutional Pediatric Case Series
Al-Huniti, Ahmad
Rathi, Nityam
Modi, Arun
Bhagavathi, Sharathkumar
Mitten, Roxane
Sharathkumar, Anjali A.
JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY, 2021, 43 (03) : E431 - E435
[2] Increased requirement for platelet transfusions concurrent with enhanced bleeding during romiplostim treatment in a patient with thrombocytopenia due to bone marrow failure
Ramin Lotfi
Peter Moeller
Mathias Schmid
Hubert Schrezenmeier
Annals of Hematology, 2011, 90 : 1357 - 1359

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