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. Author manuscript; available in PMC: 2017 Aug 10.
Published in final edited form as: Nat Rev Clin Oncol. 2016 Mar 15;13(5):273–290. doi: 10.1038/nrclinonc.2016.25

Table 3.

Clinical outcomes of CD19-targeted CAR-T-cell therapy for patients with B-ALL

Study/CAR design Conditioning/CAR-T-cell dose Patient population Clinical responses CAR-T-cell persistence
Fred Hutchinson139
FMC63-BBζ

  • Cy 60 mg/kg + Flu 25 mg/m2 daily × 3–5 (46%) or other Cy-based conditioning (54%)

  • 0.2–20 × 106 CAR T cells/kg

  • n = 24 adults with R/R disease

  • Age range: 22–71 years

  • 91% CR

  • 87% MRD (by PCR)

  • 29% sCRS

  • Persistence in responding patients >1 month

  • Improved with Flu conditioning
MD Anderson140
FMC63-28ζ

  • No conditioning

  • 1–500 × 106 CAR T cells/m2

  • n = 10

  • Post-alloHSCT uniquely, no active disease

  • 5 month DFS: 30%

  • No GVHD or sCRS

 Detectable up to 3 months
MSKCC122,134,135
SJ25-28ζ

  • Cy 1.5–3 g/m2 × 1 dose

  • 1–3 × 106 CAR T cells/kg (fractionated over 2 days)

  • Adults with R/R disease

  • Median age: 45 years (22–74 years)

  • n = 38 evaluable for safety (n = 28 evaluable for response)

  • 87% CR

  • 81% MRD-negative (by deep sequencing)

  • Median time to CR: 23 days

  • 6-month DFS: 50%

  • 6-month OS: 59%

  • 14% CD19-negative relapses

  • 23% sCRS

  • Peak day: 7–14

  • Persistence 1–3 months
NCI131,138
FMC63-28ζ

  • Cy 900 mg/m2 × 1 dose + Flu 25 mg/m2 × 3 doses

  • 1–3 × 106 CAR T cells/kg

  • Paediatric/young adults with R/R disease

  • Median age: 13 years (1–30 years)

  • n = 21 (n = 20 with B-ALL)

  • Uniquely reported intention-to-treat-analysis

  • 67% CR

  • 10-month OS: 52%

  • Two CD19-negative relapses

  • 29% sCRS

 Detectable up to day 68 (by qPCR)
UPenn/CHOP124,141,142
FMC63-BBζ

  • Variable conditioning:>50% received Flu 30 mg/m2 daily ×4 doses + Cy 500 mg/m2 daily × 2 doses

  • 0.76–20.6 × 106 CAR T cells/kg

  • Paediatric patients with R/R disease

  • Median age: 11 years

  • n = 48 evaluable

  • 94% CR

  • 82% MRD-negative (by flow cytometry)

  • 6-month DFS: 76%

  • 6-month OS: 78%

  • 67% CD19-negative relapses

  • 29% sCRS

 Range detectable:1 month–2 years (by qPCR)

alloHSCT, allogeneic haematopoietic stem cell transplantation; B-ALL, B-cell acute lymphoblastic leukaemia; CAR, chimeric antigen receptor; CHOP, Children’s Hospital of Philadelphia; CR, complete response; Cy, cyclophosphamide; DFS, disease free survival; Flu, fludarabine; Fred Hutchinson, Fred Hutchinson Cancer Center; GVHD, graft-versus-host disease; MD Anderson, MD Anderson Cancer Center; MRD, minimal residual disease; MSKCC, Memorial Sloan Kettering Cancer Center; NCI, National Cancer Institute; OS, overall survival; PCR, polymerase chain reaction; qPCR, quantitative PCR; R/R; relapsed and/or refractory; sCRS, severe cytokine-release syndrome; UPenn, University of Pennsylvania.