Table 1

Patient demographics and response. CR, complete response; PR, partial response; N/A, not available.

Subject
UPN
Age/sex
karyotype
Previous therapiesCLL tumor burden at baselineTotal
dose
of CART19
(cells/kg)
Response day
+30 (duration)
BM (study day)Blood
(study
day)
Nodes/spleen
(study day)
0165/M
normal
Fludarabine × four cycles
(2002)
Hypercellular 70% CLLN/A6.2 × 1011 to
1.0 × 1012 CLL cells
(day −37)
1.1 × 109
(1.6 × 107/kg)
CR (11+ months)
Rituximab/fludarabine ×
four cycles (2005)
2.4 × 1012 CLL cells
(day −14)
Alemtuzumab × 12 weeks
(2006)
1.7 × 1012 CLL cells
(day −1)
Rituximab (two courses,
2008 to 2009)
R-CVP × two cycles (2009)
Lenalidomide (2009)
PCR × two cycles (5/18/2010
to 6/18/2010)
Bendamustine × one cycle
(7/31/10 to 8/1/10)
pre-CART19
0277/M del
(17)(p13)*
Alemtuzumab × 16 weeks
(6/2007)
Hypercellular
>95% CLL
2.75 × 1011
CLL cells
(day −1)
1.2 × 1012 to
2.0 × 1012 CLL cells
(day −24)
5.8 × 108
(1.0 × 107/kg)
PR (7 months)
Alemtuzumab × 18 weeks
(3/2009)
3.2 × 1012 CLL cells
(day −47)
Bendamustine/rituximab:
7/1/2010 (cycle 1)
7/28/2010 (cycle 2)
8/26/2010 (cycle 3) pre-CART19
0364/M del
(17)(p13)
R-Fludarabine × two
cycles (2002)
Hypercellular
40% CLL
N/A3.3 × 1011 to
5.5 × 1011 CLL cells
(day −10)
1.4 × 107
(1.46 × 105/kg)
CR (10+ months)
R-Fludarabine × four cycles
(10/06 to 1/07)
8.8 × 1011 CLL cells
(day −1)
R-Bendamustine × one cycle
(2/09)
Bendamustine × three cycles
(3/09 to 5/09)
Alemtuzumab × 11 weeks
(12/09 to 3/10)
Pentostatin/cyclophosphamide
(9/10/10) pre-CART19

*UPN 02 karyotype [International System for Human Cytogenetic Nomenclature (ISCN)]: 45,XY,del(1)(q25),+del(1)(p13),t(2;20)(p13;q11.2),t(3;5)(p13;q35),add(9)(p22),?del(13)(q14q34),-14,del(17)(p13)[cp24].

†UPN 03 karyotype (ISCN): 46,XY,del(17)(p12)[18]/44~46,idem,der(17)t(17;21)(p11.2;q11.2)[cp4]/40~45,XY,-17[cp3].

‡See the Supplementary Material for methods of tumor burden determination.