HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR PEDIATRIC PATIENTS WITH MIXED PHENOTYPE ACUTE LEUKEMIA
(Abstract release date: 05/19/16)
EHA Library. Park J. 06/09/16; 135094; PB2194
Prof. Dr. Jeong A Park
Contributions
Contributions
Abstract
Abstract: PB2194
Type: Publication Only
Background
Mixed phenotype acute leukemia (MPAL) is a rare disease accounting for about 2-5% of all acute leukemia, and patients with MPAL have poor prognoses, particularly in patients with T/myeloid immunophenotype. However, there is no standardized treatment for MAPL and no consensus about the incorporation of hematopoietic stem cell transplantation (HCT) as an integral part of therapy. Due to its rarity, outcomes of HCT for pediatric MPAL have not been fully determined.
Aims
We investigated outcomes of HCT for pediatric patients with MPAL with multicenter study.
Methods
We retrospectively analyzed transplant outcomes for 12 pediatric patients with primary MPAL, undergoing HCT between 2001 and 2014 in 10 institutes in Youngnam province, Korea. By WHO 2008 classification, 1 (8%) had MPAL with t(v;11q23); MLL rearranged, 9 (75%) had MPAL, T/myeloid, NOS, and the remaining 2 (17%) had MPAL, B/myeloid, NOS. Before HCT, 10 patients were in first complete remission (CR), 1 patient in second CR, and one patient relapsed and had persistent disease. Donor was matched-related in 6, matched-unrelated in 2, umbilical cord blood (UCB) in 2, and autologous in 2, respectively. Conditioning regimen was busulfan, cyclophosphamide and/or melphalan based myeloablative (MA) in 10 patients and busulfan and fludarabine based nonmyeloablative (NMA) in 1 patients. One case of autologous transplant used cyclophosphamide, etoposide, cytarabine and BCNU as a conditioning regimen. For prophylaxis of GVHD following allogeneic HCT, cyclosporine and short-term methotrexate were used.
Results
Median age at the time of HCT was 9.0 years (range, 1.3-13.3 years), and median interval between diagnosis and transplantation was 6.1 months (range, 2.8-14.2 months). Cumulative incidences of neutrophil and platelet engraftment by day 28 were 100%. After median follow-up of 6.2 years (range, 0.8-17.4), 9 of 12 patients survived. Cumulative incidence of treatment-related mortality (TRM) and relapse were 9% and 35.8%. Event-free survival (EFS) and overall survival (OS) after HCT were 64.2% (50%>78%, 95% CI) and 72.7% (59%>86% 95% CI), respectively. WHO classification did not significantly affected EFS after HCT for pediatric MPAL patients (p=0.371).
Conclusion
Many studies have reported that patients with MPAL had poor prognosis, and patients with T/myeloid phenotype had a worse outcome than patients with B/myeloid phenotype. We found that WHO classification 2008 for MPAL was not significantly associated with patients’ outcome after HCT and that patients with MPAL, T/myeloid, NOS had more favorable outcome compared to historical data. Our data suggested that HCT may improve the outcome of pediatric patients with MPAL, particularly T/myeloid phenotype. This study needs to be confirmed in larger, prospective studies including more pediatric MPAL patients.
Session topic: E-poster
Keyword(s): Acute leukemia, Hematopoietic cell transplantation, Mixed lineage leukemia, Pediatric
Type: Publication Only
Background
Mixed phenotype acute leukemia (MPAL) is a rare disease accounting for about 2-5% of all acute leukemia, and patients with MPAL have poor prognoses, particularly in patients with T/myeloid immunophenotype. However, there is no standardized treatment for MAPL and no consensus about the incorporation of hematopoietic stem cell transplantation (HCT) as an integral part of therapy. Due to its rarity, outcomes of HCT for pediatric MPAL have not been fully determined.
Aims
We investigated outcomes of HCT for pediatric patients with MPAL with multicenter study.
Methods
We retrospectively analyzed transplant outcomes for 12 pediatric patients with primary MPAL, undergoing HCT between 2001 and 2014 in 10 institutes in Youngnam province, Korea. By WHO 2008 classification, 1 (8%) had MPAL with t(v;11q23); MLL rearranged, 9 (75%) had MPAL, T/myeloid, NOS, and the remaining 2 (17%) had MPAL, B/myeloid, NOS. Before HCT, 10 patients were in first complete remission (CR), 1 patient in second CR, and one patient relapsed and had persistent disease. Donor was matched-related in 6, matched-unrelated in 2, umbilical cord blood (UCB) in 2, and autologous in 2, respectively. Conditioning regimen was busulfan, cyclophosphamide and/or melphalan based myeloablative (MA) in 10 patients and busulfan and fludarabine based nonmyeloablative (NMA) in 1 patients. One case of autologous transplant used cyclophosphamide, etoposide, cytarabine and BCNU as a conditioning regimen. For prophylaxis of GVHD following allogeneic HCT, cyclosporine and short-term methotrexate were used.
Results
Median age at the time of HCT was 9.0 years (range, 1.3-13.3 years), and median interval between diagnosis and transplantation was 6.1 months (range, 2.8-14.2 months). Cumulative incidences of neutrophil and platelet engraftment by day 28 were 100%. After median follow-up of 6.2 years (range, 0.8-17.4), 9 of 12 patients survived. Cumulative incidence of treatment-related mortality (TRM) and relapse were 9% and 35.8%. Event-free survival (EFS) and overall survival (OS) after HCT were 64.2% (50%>78%, 95% CI) and 72.7% (59%>86% 95% CI), respectively. WHO classification did not significantly affected EFS after HCT for pediatric MPAL patients (p=0.371).
Conclusion
Many studies have reported that patients with MPAL had poor prognosis, and patients with T/myeloid phenotype had a worse outcome than patients with B/myeloid phenotype. We found that WHO classification 2008 for MPAL was not significantly associated with patients’ outcome after HCT and that patients with MPAL, T/myeloid, NOS had more favorable outcome compared to historical data. Our data suggested that HCT may improve the outcome of pediatric patients with MPAL, particularly T/myeloid phenotype. This study needs to be confirmed in larger, prospective studies including more pediatric MPAL patients.
Session topic: E-poster
Keyword(s): Acute leukemia, Hematopoietic cell transplantation, Mixed lineage leukemia, Pediatric
Abstract: PB2194
Type: Publication Only
Background
Mixed phenotype acute leukemia (MPAL) is a rare disease accounting for about 2-5% of all acute leukemia, and patients with MPAL have poor prognoses, particularly in patients with T/myeloid immunophenotype. However, there is no standardized treatment for MAPL and no consensus about the incorporation of hematopoietic stem cell transplantation (HCT) as an integral part of therapy. Due to its rarity, outcomes of HCT for pediatric MPAL have not been fully determined.
Aims
We investigated outcomes of HCT for pediatric patients with MPAL with multicenter study.
Methods
We retrospectively analyzed transplant outcomes for 12 pediatric patients with primary MPAL, undergoing HCT between 2001 and 2014 in 10 institutes in Youngnam province, Korea. By WHO 2008 classification, 1 (8%) had MPAL with t(v;11q23); MLL rearranged, 9 (75%) had MPAL, T/myeloid, NOS, and the remaining 2 (17%) had MPAL, B/myeloid, NOS. Before HCT, 10 patients were in first complete remission (CR), 1 patient in second CR, and one patient relapsed and had persistent disease. Donor was matched-related in 6, matched-unrelated in 2, umbilical cord blood (UCB) in 2, and autologous in 2, respectively. Conditioning regimen was busulfan, cyclophosphamide and/or melphalan based myeloablative (MA) in 10 patients and busulfan and fludarabine based nonmyeloablative (NMA) in 1 patients. One case of autologous transplant used cyclophosphamide, etoposide, cytarabine and BCNU as a conditioning regimen. For prophylaxis of GVHD following allogeneic HCT, cyclosporine and short-term methotrexate were used.
Results
Median age at the time of HCT was 9.0 years (range, 1.3-13.3 years), and median interval between diagnosis and transplantation was 6.1 months (range, 2.8-14.2 months). Cumulative incidences of neutrophil and platelet engraftment by day 28 were 100%. After median follow-up of 6.2 years (range, 0.8-17.4), 9 of 12 patients survived. Cumulative incidence of treatment-related mortality (TRM) and relapse were 9% and 35.8%. Event-free survival (EFS) and overall survival (OS) after HCT were 64.2% (50%>78%, 95% CI) and 72.7% (59%>86% 95% CI), respectively. WHO classification did not significantly affected EFS after HCT for pediatric MPAL patients (p=0.371).
Conclusion
Many studies have reported that patients with MPAL had poor prognosis, and patients with T/myeloid phenotype had a worse outcome than patients with B/myeloid phenotype. We found that WHO classification 2008 for MPAL was not significantly associated with patients’ outcome after HCT and that patients with MPAL, T/myeloid, NOS had more favorable outcome compared to historical data. Our data suggested that HCT may improve the outcome of pediatric patients with MPAL, particularly T/myeloid phenotype. This study needs to be confirmed in larger, prospective studies including more pediatric MPAL patients.
Session topic: E-poster
Keyword(s): Acute leukemia, Hematopoietic cell transplantation, Mixed lineage leukemia, Pediatric
Type: Publication Only
Background
Mixed phenotype acute leukemia (MPAL) is a rare disease accounting for about 2-5% of all acute leukemia, and patients with MPAL have poor prognoses, particularly in patients with T/myeloid immunophenotype. However, there is no standardized treatment for MAPL and no consensus about the incorporation of hematopoietic stem cell transplantation (HCT) as an integral part of therapy. Due to its rarity, outcomes of HCT for pediatric MPAL have not been fully determined.
Aims
We investigated outcomes of HCT for pediatric patients with MPAL with multicenter study.
Methods
We retrospectively analyzed transplant outcomes for 12 pediatric patients with primary MPAL, undergoing HCT between 2001 and 2014 in 10 institutes in Youngnam province, Korea. By WHO 2008 classification, 1 (8%) had MPAL with t(v;11q23); MLL rearranged, 9 (75%) had MPAL, T/myeloid, NOS, and the remaining 2 (17%) had MPAL, B/myeloid, NOS. Before HCT, 10 patients were in first complete remission (CR), 1 patient in second CR, and one patient relapsed and had persistent disease. Donor was matched-related in 6, matched-unrelated in 2, umbilical cord blood (UCB) in 2, and autologous in 2, respectively. Conditioning regimen was busulfan, cyclophosphamide and/or melphalan based myeloablative (MA) in 10 patients and busulfan and fludarabine based nonmyeloablative (NMA) in 1 patients. One case of autologous transplant used cyclophosphamide, etoposide, cytarabine and BCNU as a conditioning regimen. For prophylaxis of GVHD following allogeneic HCT, cyclosporine and short-term methotrexate were used.
Results
Median age at the time of HCT was 9.0 years (range, 1.3-13.3 years), and median interval between diagnosis and transplantation was 6.1 months (range, 2.8-14.2 months). Cumulative incidences of neutrophil and platelet engraftment by day 28 were 100%. After median follow-up of 6.2 years (range, 0.8-17.4), 9 of 12 patients survived. Cumulative incidence of treatment-related mortality (TRM) and relapse were 9% and 35.8%. Event-free survival (EFS) and overall survival (OS) after HCT were 64.2% (50%>78%, 95% CI) and 72.7% (59%>86% 95% CI), respectively. WHO classification did not significantly affected EFS after HCT for pediatric MPAL patients (p=0.371).
Conclusion
Many studies have reported that patients with MPAL had poor prognosis, and patients with T/myeloid phenotype had a worse outcome than patients with B/myeloid phenotype. We found that WHO classification 2008 for MPAL was not significantly associated with patients’ outcome after HCT and that patients with MPAL, T/myeloid, NOS had more favorable outcome compared to historical data. Our data suggested that HCT may improve the outcome of pediatric patients with MPAL, particularly T/myeloid phenotype. This study needs to be confirmed in larger, prospective studies including more pediatric MPAL patients.
Session topic: E-poster
Keyword(s): Acute leukemia, Hematopoietic cell transplantation, Mixed lineage leukemia, Pediatric
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