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Severe thrombocytopenia due to bone marrow failure in children with dyskeratosis congenita does not respond to eltrombopag treatment: case series
Division of Pediatrics, Department of Clinical Science, Intervention and Technology, Karolinska Institutet; Section of Pediatric Hematology, Immunology and HCT, Astrid Lindgren Children's Hospital.
Umeå University, Faculty of Medicine, Department of Medical Biosciences. Section of Pediatric Hematology and Oncology, Child and Adolescent Medical Center, University Hospital of Umeå, Umeå, Sweden.
Department of Clinical Immunology and Transfusion Medicine, Medical Diagnostics Center, Karolinska University Hospital, Stockholm, Sweden.
Umeå University, Faculty of Medicine, Department of Medical Biosciences. Section of Pediatric Hematology and Oncology, Child and Adolescent Medical Center, University Hospital of Umeå, Umeå, Sweden.
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2024 (English)In: Journal of Pediatric Hematology/Oncology, ISSN 1077-4114, E-ISSN 1536-3678, Vol. 46, no 1, p. 57-62Article in journal (Refereed) Published
Abstract [en]

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.

Place, publisher, year, edition, pages
Wolters Kluwer, 2024. Vol. 46, no 1, p. 57-62
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Hematology
Identifiers
URN: urn:nbn:se:umu:diva-219541DOI: 10.1097/MPH.0000000000002775PubMedID: 37885072Scopus ID: 2-s2.0-85181582874OAI: oai:DiVA.org:umu-219541DiVA, id: diva2:1828124
Available from: 2024-01-16 Created: 2024-01-16 Last updated: 2024-01-16Bibliographically approved

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Borssen, MagnusNilsson, Frans

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