Long-term outcomes of patients with refractory cytopenia of childhood under observation only.

ABSTRACT:

Abstract

Refractory cytopenia of childhood (RCC) describes an entity of well-recognized bone marrow failure defined by persistent cytopenia, dysplastic changes, and a unique histopathological pattern in the bone marrow. Although hematopoietic stem cell transplantation (HSCT) is generally indicated for patients with severe cytopenia or abnormal karyotype, a subset of patients with RCC may be candidates for an observational approach. We evaluated the long-term outcome of patients with RCC without evidence of a genetic predisposition who had a normal karyotype and had not received HSCT or immunosuppressive therapy within 2 years from diagnosis. The median age at diagnosis of the 100 patients analyzed was 10.9 years (range 1.4-17.3); 84% presented with a hypocellular bone marrow. Clona

SUBMITTER: Drexler B 

PROVIDER: S-EPMC12395045 | biostudies-literature | 2025 Aug

REPOSITORIES: biostudies-literature