Improved outcome with hematopoietic stem cell transplantation in a poor prognostic subgroup of infants with mixed-lineage-leukemia (MLL)-rearranged acute lymphoblastic leukemia: results from the Interfant-99 Study.
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Improved outcome with hematopoietic stem cell transplantation in a poor prognostic subgroup of infants with mixed-lineage-leukemia (MLL)-rearranged acute lymphoblastic leukemia: results from the Interfant-99 Study. / Mann, Georg; Attarbaschi, Andishe; Schrappe, Martin; Paola, De Lorenzo; Peters, Christina; Hann, Ian; Giulio, De Rossi; Felice, Maria; Lausen, Birgitte; Leblanc, Thierry; Szczepanski, Tomasz; Ferster, Alina; Janka-Schaub, Gritta; Rubnitz, Jeffrey; Silverman Lewis, B; Stary, Jan; Campbell, Myriam; Li, Chi Kong; Suppiah, Ram; Biondi, Andrea; Vora, Ajay; Valsecchi, Maria Grazia; Pieters, Rob; Group, Interfant-99 Study.
In: BLOOD, Vol. 116, No. 15, 15, 2010, p. 2644-2650.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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TY - JOUR
T1 - Improved outcome with hematopoietic stem cell transplantation in a poor prognostic subgroup of infants with mixed-lineage-leukemia (MLL)-rearranged acute lymphoblastic leukemia: results from the Interfant-99 Study.
AU - Mann, Georg
AU - Attarbaschi, Andishe
AU - Schrappe, Martin
AU - Paola, De Lorenzo
AU - Peters, Christina
AU - Hann, Ian
AU - Giulio, De Rossi
AU - Felice, Maria
AU - Lausen, Birgitte
AU - Leblanc, Thierry
AU - Szczepanski, Tomasz
AU - Ferster, Alina
AU - Janka-Schaub, Gritta
AU - Rubnitz, Jeffrey
AU - Silverman Lewis, B
AU - Stary, Jan
AU - Campbell, Myriam
AU - Li, Chi Kong
AU - Suppiah, Ram
AU - Biondi, Andrea
AU - Vora, Ajay
AU - Valsecchi, Maria Grazia
AU - Pieters, Rob
AU - Group, Interfant-99 Study
PY - 2010
Y1 - 2010
N2 - To define a role for hematopoietic stem cell transplantation (HSCT) in infants with acute lymphoblastic leukemia and rearrangements of the mixed-lineage-leukemia gene (MLL(+)), we compared the outcome of MLL(+) patients from trial Interfant-99 who either received chemotherapy only or HSCT. Of 376 patients with a known MLL status in the trial, 297 (79%) were MLL(+). Among the 277 of 297 MLL(+) patients (93%) in first remission (CR), there appeared to be a significant difference in disease-free survival (adjusted by waiting time to HSCT) between the 37 (13%) who received HSCT and the 240 (87%) who received chemotherapy only (P = .03). However, the advantage was restricted to a subgroup with 2 additional unfavorable prognostic features: age less than 6 months and either poor response to steroids at day 8 or leukocytes more than or equal to 300 g/L. Ninety-seven of 297 MLL(+) patients (33%) had such high-risk criteria, with 87 achieving CR. In this group, HSCT was associated with a 64% reduction in the risk of failure resulting from relapse or death in CR (hazard ratio = 0.36, 95% confidence interval, 0.15-0.86). In the remaining patients, there was no advantage for HSCT over chemotherapy only. In summary, HSCT seems to be a valuable option for a subgroup of infant MLL(+) acute lymphoblastic leukemia carrying further poor prognostic factors. The trial was registered at www.clinicaltrials.gov as #NCT00015873 and at www.controlled-trials.com as #ISRCTN24251487.
AB - To define a role for hematopoietic stem cell transplantation (HSCT) in infants with acute lymphoblastic leukemia and rearrangements of the mixed-lineage-leukemia gene (MLL(+)), we compared the outcome of MLL(+) patients from trial Interfant-99 who either received chemotherapy only or HSCT. Of 376 patients with a known MLL status in the trial, 297 (79%) were MLL(+). Among the 277 of 297 MLL(+) patients (93%) in first remission (CR), there appeared to be a significant difference in disease-free survival (adjusted by waiting time to HSCT) between the 37 (13%) who received HSCT and the 240 (87%) who received chemotherapy only (P = .03). However, the advantage was restricted to a subgroup with 2 additional unfavorable prognostic features: age less than 6 months and either poor response to steroids at day 8 or leukocytes more than or equal to 300 g/L. Ninety-seven of 297 MLL(+) patients (33%) had such high-risk criteria, with 87 achieving CR. In this group, HSCT was associated with a 64% reduction in the risk of failure resulting from relapse or death in CR (hazard ratio = 0.36, 95% confidence interval, 0.15-0.86). In the remaining patients, there was no advantage for HSCT over chemotherapy only. In summary, HSCT seems to be a valuable option for a subgroup of infant MLL(+) acute lymphoblastic leukemia carrying further poor prognostic factors. The trial was registered at www.clinicaltrials.gov as #NCT00015873 and at www.controlled-trials.com as #ISRCTN24251487.
KW - Humans
KW - Risk Factors
KW - Treatment Outcome
KW - Age Factors
KW - Prognosis
KW - Survival Analysis
KW - Disease-Free Survival
KW - Leukocyte Count
KW - Hematopoietic Stem Cell Transplantation
KW - Gene Rearrangement
KW - Myeloid-Lymphoid Leukemia Protein genetics
KW - Precursor Cell Lymphoblastic Leukemia-Lymphoma blood
KW - Humans
KW - Risk Factors
KW - Treatment Outcome
KW - Age Factors
KW - Prognosis
KW - Survival Analysis
KW - Disease-Free Survival
KW - Leukocyte Count
KW - Hematopoietic Stem Cell Transplantation
KW - Gene Rearrangement
KW - Myeloid-Lymphoid Leukemia Protein genetics
KW - Precursor Cell Lymphoblastic Leukemia-Lymphoma blood
M3 - SCORING: Zeitschriftenaufsatz
VL - 116
SP - 2644
EP - 2650
JO - BLOOD
JF - BLOOD
SN - 0006-4971
IS - 15
M1 - 15
ER -
