HSCT

A report from the European Working Group on Childhood Myelodysplastic Syndromes included 100 transplant recipients at multiple centers treated with a common preparative regimen of busulfan, cyclophosphamide, and melphalan, with or without antithymocyte globulin. Recipients had been treated with varying degrees of pretransplant chemotherapy or differentiating agents, and some patients had a splenectomy.[7]

A report from the European Working Group on Childhood Myelodysplastic Syndromes included 100 transplant recipients at multiple centers treated with a common preparative regimen of busulfan, cyclophosphamide, and melphalan, with or without antithymocyte globulin. Recipients had been treated with varying degrees of pretransplant chemotherapy or differentiating agents, and some patients had a splenectomy.[7]

The 5-year EFS rate was 55% for children with JMML who underwent HSCT using HLA-identical matched family donor cells and 49% for children with JMML who underwent HSCT using unrelated donor cells.

A report from the European Working Group on Childhood Myelodysplastic Syndromes included 100 transplant recipients at multiple centers treated with a common preparative regimen of busulfan, cyclophosphamide, and melphalan, with or without antithymocyte globulin. Recipients had been treated with varying degrees of pretransplant chemotherapy or differentiating agents, and some patients had a splenectomy.[7]

The multivariate analysis showed no effect on survival of previous acute myeloid leukemia–like chemotherapy versus low-dose chemotherapy or no chemotherapy.

A report from the European Working Group on Childhood Myelodysplastic Syndromes included 100 transplant recipients at multiple centers treated with a common preparative regimen of busulfan, cyclophosphamide, and melphalan, with or without antithymocyte globulin. Recipients had been treated with varying degrees of pretransplant chemotherapy or differentiating agents, and some patients had a splenectomy.[7]

No effect on survival was observed for splenectomy pretransplant or difference in spleen size.

A report from the European Working Group on Childhood Myelodysplastic Syndromes included 100 transplant recipients at multiple centers treated with a common preparative regimen of busulfan, cyclophosphamide, and melphalan, with or without antithymocyte globulin. Recipients had been treated with varying degrees of pretransplant chemotherapy or differentiating agents, and some patients had a splenectomy.[7]

No difference in outcomes was found based on related versus unrelated donors.

A report from the European Working Group on Childhood Myelodysplastic Syndromes included 100 transplant recipients at multiple centers treated with a common preparative regimen of busulfan, cyclophosphamide, and melphalan, with or without antithymocyte globulin. Recipients had been treated with varying degrees of pretransplant chemotherapy or differentiating agents, and some patients had a splenectomy.[7]

Only age older than 4 years and female sex were shown to be poor prognostic factors for outcome and increased risk of relapse (relative risk [RR], 2.24 [1.07–4.69]; P = .032 for older age; RR, 2.22 [1.09–4.50]; P = .028 for females).[7]

In one study, cord blood transplant produced the following results:[10][Level of evidence C2]

In one study, cord blood transplant produced the following results:[10][Level of evidence C2]

The 5-year disease-free survival rate was 44%.

In one study, cord blood transplant produced the following results:[10][Level of evidence C2]

Outcomes were improved in children younger than 1.4 years at diagnosis, those with nonmonosomy 7 karyotype, and those receiving 5/6 to 6/6 HLA-matched cord units.

In one study, cord blood transplant produced the following results:[10][Level of evidence C2]

This suggests that cord blood can provide an additional donor pool for this group of children.

The use of reduced-intensity preparative regimens to decrease the adverse side effects of transplant have also been reported in small numbers of patients, generally for patients ineligible for myeloablative HSCT.[11,12]

The use of reduced-intensity preparative regimens to decrease the adverse side effects of transplant have also been reported in small numbers of patients, generally for patients ineligible for myeloablative HSCT.[11,12]

The COG conducted a randomized trial in children with JMML that compared a standard-intensity preparative regimen (busulfan/cyclophosphamide/melphalan) with a reduced-intensity regimen (busulfan/fludarabine).[13]

The use of reduced-intensity preparative regimens to decrease the adverse side effects of transplant have also been reported in small numbers of patients, generally for patients ineligible for myeloablative HSCT.[11,12] › The COG conducted a randomized trial in children with JMML that compared a standard-intensity preparative regimen (busulfan/cyclophosphamide/melphalan) with a reduced-intensity regimen (busulfan/fludarabine).[13]

The trial closed to enrollment early when an interim analysis revealed a higher frequency of relapse/disease persistence (7 of 9 patients) in children who received the reduced-intensity regimen than in children who received the standard-intensity regimen (1 of 6 patients).