Table 1.

Summary of HSCT indications and outcomes for ALL in CR1 on pediatric trials

TrialYears of enrollmentRegionCriteria for HSCT in CR1N*OutcomesComments
AALL00316  2002-2006 USA Hypodiploidy, MLL rearrangement plus SER, induction failure 30 Hypodiploidy: 4-year DFS for chemotherapy, 50% ± 11% vs HSCT, 62% ± 14% (P = .65).
Induction failure: 4-year DFS for chemotherapy, 44% ± 23%; chemotherapy vs HSCT, 75% ± 19% (P = .14) 		Ph+ ALL excluded from analysis; nonrandomized study 
Total Therapy 1312,49  1991-1998 USA Ph+ ALL, induction failure 57 Combined Total Therapy 13/14 trials 5-year OS: 28% (95% CI, 17%-40%) Nonrandomized study 
Total Therapy 144,12  1998-1999 USA Ph+ ALL, induction failure Nonrandomized study; trial terminated early because of excess toxicity 
Total Therapy 1512,50  2000-2007 USA Ph+ ALL, induction failure; MRD ≥1% after 6 weeks of induction 37 5-year OS: 65% (95% CI, 46%-78%) Nonrandomized study 
ALL BFM-9051  1990-1995 Europe Induction failure, Ph+ ALL or PPR and T-ALL; myeloid marker BFM-RF ≥1.7 or t(4;11) 35 NR Nonrandomized study 
ALL BFM-9511  1995-2000 Europe Induction failure, Ph+ ALL, t(4;11), PPR and T-ALL or WBC ≥100 × 109/L; only patients with matched related donor underwent HSCT 77 5-year DFS for chemotherapy: 40.6% (SE, 3.1%); chemotherapy vs HSCT, 56.7% (SE, 5.7%) (hazard ratio, 0.67; 95% CI, 0.46-0.99; P = .02) Patients were randomly assigned between chemotherapy and related-donor HSCT 
AEIOP-BFM ALL 200024  2000-2006 Europe 

  • (1) PPR and T-cell ALL or WBC ≥100 ×109/L or pro-B ALL or MRD ≥10−2 at day 33;

  • (2) MRD ≥10−3 at day 78 or t(4;11) and PGR;

  • (3) induction failure or MRD>10−2 at day 78 or t(4;11) and PPR

 		81 5-year DFS for chemotherapy vs HSCT: (1) 67.7% (SE=6.3) vs 83.3% (SE=10.8; p=0.31); (2) 47.2% (SE=6.6) vs 51.1% (SE=9.6; p=0.74); (3) 54.7% (SE=13.6) vs 50.5% (SE=8; p=0.79) Ph+ ALL excluded; nonrandomized study; required matched donor (related for subgroups 1/2) for HSCT 
NOPHO ALL-925  1992-2001 Scandinavia No uniform criteria 57 NR Nonrandomized study 
NOPHO ALL-20005  2002-2007 Scandinavia Induction failure, Ph+ ALL, WBC ≥200 × 109/L, MLL rearrangement and age older than 10 years, hypodiploidy (<34); optional: MRD ≥10−3 at 3 months 62 NR Nonrandomized study 
NOPHO ALL-200825  2008-2016 Scandinavia Induction failure, MRD ≥0.1% at day 79; optional: hypodiploidy (<44) with good response 71 DFS, 79.1% (95% CI, 69.8%-89.6%) at median follow-up of 5.5 years since HSCT Ph+ ALL patients excluded; nonrandomized study 
TrialYears of enrollmentRegionCriteria for HSCT in CR1N*OutcomesComments
AALL00316  2002-2006 USA Hypodiploidy, MLL rearrangement plus SER, induction failure 30 Hypodiploidy: 4-year DFS for chemotherapy, 50% ± 11% vs HSCT, 62% ± 14% (P = .65).
Induction failure: 4-year DFS for chemotherapy, 44% ± 23%; chemotherapy vs HSCT, 75% ± 19% (P = .14) 		Ph+ ALL excluded from analysis; nonrandomized study 
Total Therapy 1312,49  1991-1998 USA Ph+ ALL, induction failure 57 Combined Total Therapy 13/14 trials 5-year OS: 28% (95% CI, 17%-40%) Nonrandomized study 
Total Therapy 144,12  1998-1999 USA Ph+ ALL, induction failure Nonrandomized study; trial terminated early because of excess toxicity 
Total Therapy 1512,50  2000-2007 USA Ph+ ALL, induction failure; MRD ≥1% after 6 weeks of induction 37 5-year OS: 65% (95% CI, 46%-78%) Nonrandomized study 
ALL BFM-9051  1990-1995 Europe Induction failure, Ph+ ALL or PPR and T-ALL; myeloid marker BFM-RF ≥1.7 or t(4;11) 35 NR Nonrandomized study 
ALL BFM-9511  1995-2000 Europe Induction failure, Ph+ ALL, t(4;11), PPR and T-ALL or WBC ≥100 × 109/L; only patients with matched related donor underwent HSCT 77 5-year DFS for chemotherapy: 40.6% (SE, 3.1%); chemotherapy vs HSCT, 56.7% (SE, 5.7%) (hazard ratio, 0.67; 95% CI, 0.46-0.99; P = .02) Patients were randomly assigned between chemotherapy and related-donor HSCT 
AEIOP-BFM ALL 200024  2000-2006 Europe 

  • (1) PPR and T-cell ALL or WBC ≥100 ×109/L or pro-B ALL or MRD ≥10−2 at day 33;

  • (2) MRD ≥10−3 at day 78 or t(4;11) and PGR;

  • (3) induction failure or MRD>10−2 at day 78 or t(4;11) and PPR

 		81 5-year DFS for chemotherapy vs HSCT: (1) 67.7% (SE=6.3) vs 83.3% (SE=10.8; p=0.31); (2) 47.2% (SE=6.6) vs 51.1% (SE=9.6; p=0.74); (3) 54.7% (SE=13.6) vs 50.5% (SE=8; p=0.79) Ph+ ALL excluded; nonrandomized study; required matched donor (related for subgroups 1/2) for HSCT 
NOPHO ALL-925  1992-2001 Scandinavia No uniform criteria 57 NR Nonrandomized study 
NOPHO ALL-20005  2002-2007 Scandinavia Induction failure, Ph+ ALL, WBC ≥200 × 109/L, MLL rearrangement and age older than 10 years, hypodiploidy (<34); optional: MRD ≥10−3 at 3 months 62 NR Nonrandomized study 
NOPHO ALL-200825  2008-2016 Scandinavia Induction failure, MRD ≥0.1% at day 79; optional: hypodiploidy (<44) with good response 71 DFS, 79.1% (95% CI, 69.8%-89.6%) at median follow-up of 5.5 years since HSCT Ph+ ALL patients excluded; nonrandomized study 

Berlin-Frankfurt-Muenster risk factor (BFM-RF) calculated by 0.2 × log (peripheral blood blasts per μL + 1) + 0.06 × liver size in centimeters below the costal margin + 0.04 × spleen size in centimeters below the costal margin.

NR, not reported; NOPHO, optional indication for HSCT; OS, overall survival; PGR, prednisone good; PPR, poor prednisone response; SE, standard error; SER, slow early response; WBC, white blood cell count.

*

Number of patients who underwent HSCT.

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