Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®): Treatment - Health Pro
Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®): Treatment - Health Professional Information [NCI]-Myelodysplastic Syndromes
Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®): Treatment - Health Professional Information [NCI] Guide
A significant issue to consider for these results is that the subtype refractory anemia with excess blasts in transformation is likely to represent patients with overt AML, while refractory anemia and refractory anemia with excess blasts represent MDS. The WHO classification has now omitted the category of refractory anemia with excess blasts in transformation, concluding that this entity was essentially AML. The optimal therapy for patients with refractory anemia/refractory anemia with excess blasts without an HLA-MFD is unknown. Some of these patients require no therapy for years and have indolent diseases. Because failure rates after HSCT are lower in this group, strong consideration should be given for transplantation, especially when a 5/6 or 6/6 HLA-MFD is available. However, alternative forms of HSCT, utilizing matched unrelated donor cord blood, should be considered when treatment is required, as is usually the case in patients with severe symptomatic cytopenias.[29,32] The 8-year DFS for children with various stages of MDS transplanted with either HLA matched or mismatched unrelated donor transplants has been reported to be 65% and 40%, respectively.[32][Level of evidence: 3iiiDii] A 3-year DFS of 50% was reported with the use of unrelated cord blood donor transplants for children with MDS, when the transplants were done after 2001.[39][Level of evidence: 3iiiDiii]
Because MDS in children is often associated with inherited predisposition syndromes, reports of transplantation in small numbers of patients have been reported. For example, in patients with Fanconi anemia and AML or advanced MDS, the 5-year overall survival (OS) has been reported to be 33% to 55%.[40,41][Level of evidence: 3iiiA] Second transplants have also been used in pediatric patients with MDS/MPD who relapse or suffer graft failure. The 3-year OS was 33% for those retransplanted for relapse and 57% for those transplanted for initial graft failure.[42][Level of evidence: 3iiiA]
For patients with clinically significant cytopenias, supportive care, including transfusions and prophylactic antibiotics, can be considered. In addition, the use of hematopoietic growth factors can improve the hematopoietic status, but there remains some concern that such treatment could accelerate conversion to AML.[43] Steroid therapy has also been used, including glucocorticoids and androgens, with mixed results.[44] Treatments directed toward scavenging free oxygen radicals with amifostine [45,46] or the use of differentiation-promoting retinoids,[47] DNA methylation inhibitors (e.g., azacytidine and decitabine), and histone deacetylase inhibitors, have all shown some response, but no definitive trials in children with MDS have been reported. Azacytidine has been by the U.S. Food and Drug Administration (FDA) -approved for the treatment of MDS in adults based on randomized studies.[48] Agents, such as lenalidomide, an analog of thalidomide, have been tested based on findings that demonstrated increased activity in the bone marrow of patients with MDS. Lenalidomide has shown most efficacy in patients with 5q- syndrome, especially those with thrombocytosis, and is now FDA-approved for use in this group.[49] Immunosuppression with antithymocyte globulin and/or cyclosporine has also been reported.[49,50]
Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Myelodysplastic Syndromes
Childhood Acute Myeloid Leukemia/Other Myeloid Malignancies Treatment (PDQ®): Treatment - Health Professional Information [NCI] Guide
- General Information
- Classification of Pediatric Myeloid Malignancies
- Treatment Overview for Acute Myeloid Leukemia (AML)
- Treatment of Newly Diagnosed AML
- Postremission Therapy for AML
- Acute Promyelocytic Leukemia
- Children with Down Syndrome
- Myelodysplastic Syndromes
- Therapy-Related AML / Myelodysplastic Syndromes
- Juvenile Myelomonocytic Leukemia
- Chronic Myelogenous Leukemia
- Recurrent Childhood AML and Other Myeloid Malignancies
- Survivorship and Adverse Late Sequelae
- Changes to This Summary (08 / 15 / 2014)
- About This PDQ Summary
- Get More Information From NCI
A significant issue to consider for these results is that the subtype refractory anemia with excess blasts in transformation is likely to represent patients with overt AML, while refractory anemia and refractory anemia with excess blasts represent MDS. The WHO classification has now omitted the category of refractory anemia with excess blasts in transformation, concluding that this entity was essentially AML. The optimal therapy for patients with refractory anemia/refractory anemia with excess blasts without an HLA-MFD is unknown. Some of these patients require no therapy for years and have indolent diseases. Because failure rates after HSCT are lower in this group, strong consideration should be given for transplantation, especially when a 5/6 or 6/6 HLA-MFD is available. However, alternative forms of HSCT, utilizing matched unrelated donor cord blood, should be considered when treatment is required, as is usually the case in patients with severe symptomatic cytopenias.[29,32] The 8-year DFS for children with various stages of MDS transplanted with either HLA matched or mismatched unrelated donor transplants has been reported to be 65% and 40%, respectively.[32][Level of evidence: 3iiiDii] A 3-year DFS of 50% was reported with the use of unrelated cord blood donor transplants for children with MDS, when the transplants were done after 2001.[39][Level of evidence: 3iiiDiii]
Because MDS in children is often associated with inherited predisposition syndromes, reports of transplantation in small numbers of patients have been reported. For example, in patients with Fanconi anemia and AML or advanced MDS, the 5-year overall survival (OS) has been reported to be 33% to 55%.[40,41][Level of evidence: 3iiiA] Second transplants have also been used in pediatric patients with MDS/MPD who relapse or suffer graft failure. The 3-year OS was 33% for those retransplanted for relapse and 57% for those transplanted for initial graft failure.[42][Level of evidence: 3iiiA]
For patients with clinically significant cytopenias, supportive care, including transfusions and prophylactic antibiotics, can be considered. In addition, the use of hematopoietic growth factors can improve the hematopoietic status, but there remains some concern that such treatment could accelerate conversion to AML.[43] Steroid therapy has also been used, including glucocorticoids and androgens, with mixed results.[44] Treatments directed toward scavenging free oxygen radicals with amifostine [45,46] or the use of differentiation-promoting retinoids,[47] DNA methylation inhibitors (e.g., azacytidine and decitabine), and histone deacetylase inhibitors, have all shown some response, but no definitive trials in children with MDS have been reported. Azacytidine has been by the U.S. Food and Drug Administration (FDA) -approved for the treatment of MDS in adults based on randomized studies.[48] Agents, such as lenalidomide, an analog of thalidomide, have been tested based on findings that demonstrated increased activity in the bone marrow of patients with MDS. Lenalidomide has shown most efficacy in patients with 5q- syndrome, especially those with thrombocytosis, and is now FDA-approved for use in this group.[49] Immunosuppression with antithymocyte globulin and/or cyclosporine has also been reported.[49,50]