Outcomes of children, adolescents, and young adults following allogeneic stem cell transplantation for secondary acute myeloid leukemia and myelodysplastic syndromes—The MD Anderson Cancer Center experience

Abstract

We conducted a retrospective analysis of outcomes for children and young adults with sAML/sMDS who underwent HSCT at our institution. Thirty-two patients (median age 20 years) with sAML (n=24) and sMDS (n=8) received HSCT between 1990 and 2013. The median time from sAML/sMDS diagnosis to HSCT was 4.1 months (range: 1.2-27.2 months). The transplant regimens were primarily busulfan based (n=19). BM was the primary donor source (n=15). Eleven recipients were transplanted with residual disease. At a median follow-up of 62.3 months (range: 0.4-250.9 months), 14 patients had disease recurrence. Acute GVHD, grade III/IV, occurred in three patients. Causes of death were as follows: disease relapse (n=12), infection (n=2), pneumonia (n=1), pulmonary hemorrhage (n=1), acute GVHD (n=1), and graft failure (n=1). A PS of ≥90% at the time of HSCT had a significant impact on PFS (P=.02). Patients achieving pretransplant primary CR (n=8) and those with sMDS and RA (n=6) had prolonged PFS (P=.04). On multivariate analysis, shorter time to transplantation (≤6 months from diagnosis of sAML/sMDS) was associated with superior OS (P=.0018) and PFS (P=.0005).

Treatment of recurrent pediatric myelodysplastic syndrome post hematopoietic stem cell transplantation

“…Survival is dismal for the 40%–60% of children with myelodysplastic syndrome (MDS) who relapse post allogeneic hematopoietic cell transplantation (HCT) 1–3 . Strategies to decrease relapse risk includes use of cytoreduction prior to HCT or maintenance treatment after HCT, data on the utility of these approaches remains limited 4–12 .…”

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Survival is dismal for the 40%-60% of children with myelodysplastic syndrome (MDS) who relapse post allogeneic hematopoietic cell transplantation (HCT). [1][2][3] Strategies to decrease relapse risk includes use of cytoreduction prior to HCT or maintenance treatment after HCT, data on the utility of these approaches remains limited. [4][5][6][7][8][9][10][11][12] Rapid withdrawal of immune suppression or use of donor lymphocyte infusion (DLI) can enhance the graft versus leukemia effect and achieve disease control in some cases.

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Treatment of Recurrent Pediatric Myelodysplastic Syndrome Post Hematopoietic Stem Cell Transplantation

“…Survival is dismal for the 40-60% of children with MDS who relapse post allogeneic HCT 1,2,3 . Strategies to decrease relapse risk include use of cytoreduction prior to HCT or maintenance treatment after HCT, data on the utility of these approaches remains limited [4][5][6][7][8][9][10][11][12] .…”