TY - JOUR
T1 - Comparison of melphalan- And busulfan-based myeloablative conditioning in children undergoing allogeneic transplantation for acute myeloid leukemia or myelodysplasia
AU - Oshrine, Benjamin
AU - Adams, Lauren
AU - Nguyen, Anh Thy H.
AU - Amankwah, Ernest
AU - Shyr, David
AU - Hale, Gregory
AU - Petrovic, Aleksandra
PY - 2020/6/1
Y1 - 2020/6/1
N2 - Background: The optimal conditioning regimen for alloHCT in children with myeloid malignancies remains undefined. Procedure: We performed a retrospective review of children undergoing alloHCT for AML and MDS over a 10-year period (2008-2018) at our institution, comparing the outcomes of recipients of either a myeloablative busulfan- or reduced toxicity mel/thio-based conditioning regimen. Results: A total of 49 patients underwent alloHCT for AML/MDS (mel/thio, N = 21; busulfan, N = 28). Mel/thio recipients were selected due to pretransplant comorbidities. Recipients of mel/thio were more likely to have t-AML, and less likely to have MRD <0.1% at the time of alloHCT (57.1% vs 82.1%). Graft failure was more common in busulfan recipients; engraftment kinetics were similar between groups. Sinusoidal obstructive syndrome was diagnosed in 21% of busulfan and no mel/thio recipients (P =.03). One patient in each group died from TRM. Relapse incidence was comparable (mel/thio—29% vs busulfan—32%); however, relapse occurred significantly later in recipients of mel/thio conditioning (median d + 396 vs d + 137; P =.01). As a result, there was a trend toward improved OS at 1 and 3 years in mel/thio recipients (95% vs 74%, P =.06; and 75% vs 50%, P =.11; respectively). Conclusion: In our single institution, when compared to myeloablative busulfan-based conditioning, use of a mel/thio-based reduced toxicity regimen resulted in comparable outcomes, despite higher risk patient and disease characteristics. Mel/thio recipients had both more comorbidities and higher risk disease profile, which did not translate into higher rates of either TRM or relapse.
AB - Background: The optimal conditioning regimen for alloHCT in children with myeloid malignancies remains undefined. Procedure: We performed a retrospective review of children undergoing alloHCT for AML and MDS over a 10-year period (2008-2018) at our institution, comparing the outcomes of recipients of either a myeloablative busulfan- or reduced toxicity mel/thio-based conditioning regimen. Results: A total of 49 patients underwent alloHCT for AML/MDS (mel/thio, N = 21; busulfan, N = 28). Mel/thio recipients were selected due to pretransplant comorbidities. Recipients of mel/thio were more likely to have t-AML, and less likely to have MRD <0.1% at the time of alloHCT (57.1% vs 82.1%). Graft failure was more common in busulfan recipients; engraftment kinetics were similar between groups. Sinusoidal obstructive syndrome was diagnosed in 21% of busulfan and no mel/thio recipients (P =.03). One patient in each group died from TRM. Relapse incidence was comparable (mel/thio—29% vs busulfan—32%); however, relapse occurred significantly later in recipients of mel/thio conditioning (median d + 396 vs d + 137; P =.01). As a result, there was a trend toward improved OS at 1 and 3 years in mel/thio recipients (95% vs 74%, P =.06; and 75% vs 50%, P =.11; respectively). Conclusion: In our single institution, when compared to myeloablative busulfan-based conditioning, use of a mel/thio-based reduced toxicity regimen resulted in comparable outcomes, despite higher risk patient and disease characteristics. Mel/thio recipients had both more comorbidities and higher risk disease profile, which did not translate into higher rates of either TRM or relapse.
KW - acute myeloid leukemia
KW - hematopoietic cell transplantation
KW - myelodysplastic syndrome
KW - pediatric
KW - reduced toxicity conditioning
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U2 - 10.1111/petr.13672
DO - 10.1111/petr.13672
M3 - Article
C2 - 32068340
AN - SCOPUS:85079700131
SN - 1397-3142
VL - 24
JO - Pediatric transplantation
JF - Pediatric transplantation
IS - 4
M1 - e13672
ER -