Effective treatment of refractory acquired pure red blood cell aplasia with eltrombopag and sirolimus: a case report.
acquired pure red cell aplasia
eltrombopag
refractory
sirolimus
Journal
Therapeutic advances in hematology
ISSN: 2040-6207
Titre abrégé: Ther Adv Hematol
Pays: England
ID NLM: 101549589
Informations de publication
Date de publication:
2020
2020
Historique:
received:
26
03
2020
accepted:
11
06
2020
entrez:
1
8
2020
pubmed:
1
8
2020
medline:
1
8
2020
Statut:
epublish
Résumé
Acquired pure red cell aplasia (aPRCA) is a kind of anemia characterized by severe reticulocytopenia and reduced bone marrow erythroblastic cells. For patients who are refractory to the first-line therapy (cyclosporin A with/without glucocorticoids), second-line therapy is considered less effective. We report on a patient with primary aPRCA who was refractory to cyclosporin A, glucocorticoids, and several second-line regimens. The patient was treated with sirolimus for 10 months with no improvement in hemoglobin but complete response was achieved after adding eltrombopag at a dosage of 25 mg/day. Eltrombopag was well tolerated with no evidence of clonal evolution at the end of follow up. This case provided a new attempt at treating patients with refractory/relapse aPRCA with eltrombopag, probably in combination with sirolimus.
Identifiants
pubmed: 32733663
doi: 10.1177/2040620720940144
pii: 10.1177_2040620720940144
pmc: PMC7372523
doi:
Types de publication
Case Reports
Langues
eng
Pagination
2040620720940144Informations de copyright
© The Author(s), 2020.
Déclaration de conflit d'intérêts
Conflict of interest statement: The authors declare that there is no conflict of interest.
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