Complete resolution of primary myelofibrosis in an infant with steroids and hydroxyurea.
BMJ Case Rep
; 16(11)2023 Nov 22.
Article
em En
| MEDLINE
| ID: mdl-37993140
ABSTRACT
Paediatric primary myelofibrosis (PMF) is exceedingly rare and distinct compared with adult PMF. It is characterised by peripheral blood cytopenias, leucoerythroblastosis, reticulin fibrosis, extramedullary haematopoiesis and hepatosplenomegaly. In the absence of laid down diagnostic criteria, the diagnosis is largely of exclusion. Though early haematological stem cell transplant (HSCT) remains the treatment of choice, spontaneous remission or remission with steroids and/or cytoreductive agents is described in around 20% of cases of paediatric PMF. Moreover, HSCT in paediatric PMF is associated with high mortality (30%-45%). Therefore, it may be prudent to consider a trial of steroids and/or cytoreductive agents in all transfusion-dependent paediatric PMF while considering HSCT and ongoing bone marrow donor search. We describe one such infant with PMF who had complete remission of clinical and haematological parameters with a combination therapy of steroids and hydroxyurea.
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Base de dados:
MEDLINE
Assunto principal:
Trombocitopenia
/
Mielofibrose Primária
Idioma:
En
Ano de publicação:
2023
Tipo de documento:
Article