Studies have attempted to retrospectively classify and analyze the outcome of children with myelodysplastic syndromes (MDS).[1,2] This continues to be problematic. The French-American-British (FAB) classification of adult MDS is only partially helpful in the categorization of children with MDS. Children with MDS present with FAB subtypes of refractory anemia (RA), RA with excess blasts (RAEB), and RA with excess blasts in transformation (RAEB-T). Juvenile myelomonocytic leukemia and monosomy 7 are discussed separately.
The optimal therapy for childhood MDS is controversial. The Children's Cancer Group 2891 trial accrued patients between 1989 and 1995, including children with MDS. There were 77 patients with RA (2), RAEB (33), RAEB-T (26), or acute myeloid leukemia (AML) with antecedent MDS (16) who were enrolled and randomized to standard or intensively timed induction. Subsequently, patients were allocated to allogeneic hematopoietic stem cell transplantation (HSCT) if there was a suitable family donor, or randomized to autologous HSCT or chemotherapy. Patients with RA/RAEB had a poor remission rate (45%), and those with RAEB-T (69%) or AML with history of MDS (81%) had similar remission rates compared with de novo AML (77%). Six-year survival was poor for those with RA/RAEB (28%) and RAEB-T (30%). Patients with AML and antecedent MDS had a similar outcome to those with de novo AML (50% survival compared with 45%). Allogeneic HSCT appeared to improve survival at a marginal level of significance (P = .08). Based on analysis of these data and the literature, the authors conclude that children with a history of MDS who present with AML (excluding those with monosomy 7) and many of those with RAEB-T do as well with AML therapy at diagnosis as AML patients. For patients who achieve remission and for whom there is no matched-family donor (MFD), it is unclear whether aggressive continuation of chemotherapy or alternative donor stem cell transplant is optimum therapy. Children with RA/RAEB as well as patients with AML have a low response rate to AML induction therapy. Because failure rates after HSCT are lower in this group when treated at diagnosis, strong consideration should be given for such treatment, especially when a 5/6 or 6/6 HLA-MFD is available. The optimum therapy for patients with RA/RAEB without MFDs is unknown. Some of these patients require no therapy for years and have indolent diseases. However, alternative forms of HSCT, utilizing matched unrelated donor cord blood, should be considered when treatment is required, usually for severe cytopenia. An analysis of 37 children with MDS treated on Berlin-Frankfurt-Munster AML protocols 83, 87, and 93 confirmed the induction response of 74% for patients with RAEB-T, and suggested that transplantation was beneficial.
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The following are examples of national and/or institutional clinical trials that are currently being conducted.
The use of a variety of inhibitors of DNA methylation and histone deacetylase inhibitors, as well as other therapies designed to induce differentiation, are being studied in both young and older adults with MDS.[6,7,8]