Comparison of clinical trial versus non-clinical trial treatment outcomes of childhood acute lymphoblastic leukemia using comparable regimens

Figures & data

Table 1 Demographics and clinical features of patients treated on CCG-1991 (regimen OD) versus comparable regimen from the CCG-1991 clinical trial

	CCG-1991		P-value
	Current study	Matloub et al. study*
	CCG-1991 (regimen OD)	CCG-1991 (equivalent regimen)
	n (%)	n (%)
Number of patients	120	1036^†
Age (years)
1–1.9	13 (10.8)	85 (8.2)	0.401
2–5.9	78 (65.0)	733 (70.8)	0.231
6–9.9	29 (24.2)	218 (21.0)	0.501
Sex
Female	54 (45.0)	475 (45.8)	0.936
Male	66 (55.0)	561 (54.2)
Ethnicity	Arabs/Middle Eastern	Heterogeneous
WBC count^‡
WBC <20 × 10^9/l	102 (85.0)	856 (82.6)	0.660
WBC ≥20 × 10^9/l	18 (15.0)	176 (17.0)
CNS status^‡
CNS1	113 (94.2)	799 (77.1)	0.437
CNS2	7 (5.8)	32 (3.1)

*Data extrapolated from Matloub et al.³

^†Number of patients treated on the oral methotrexate regimens from the original CCG-1991.

^‡Percentages do not add up to 100%.

Figure 1 OS and EFS of: (A) SR patients treated on CCG-1991 (regimen OD), and (B) HR patients treated on CCG-1961 (regimen D).

Table 2 Comparison of outcomes of patients treated on CCG-1991 (regimen OD) versus comparable regimen from the CCG-1991 clinical trial

	CCG-1991		P-value
	Current study	Matloub et al. study*
	CCG-1991 (regimen OD)	CCG-1991 (equivalent regimen)
Number of patients	120	1036^†
Study design	Retrospective	Prospective
	Single center	Multicenter
Study period	January 2001–December 2007	June 2000–February 2005
Five-year OS	89.0 ± 2.9%	96.0 ± 0.9%	SIG
Five-year EFS	82.3 ± 3.5%	88.7 ± 1.4%	SIG
Relapse/events	19 (15.8%)	96 (9.3%)	0.034
Isolated BM relapse	9 (7.5%)	43 (4.2%)	0.149
Isolated CNS relapse	6 (5.0%)	26 (2.5%)	0.134
BM + CNS	3 (2.5%)	10 (0.1%)	0.144
BM + EM	0	4 (0.4%)	1.000
Testicular	1 (0.8%)	7 (0.7%)	0.585
Other EM	0	6 (0.6%)	1.000
Death in remission	2 (1.7%)	5 (0.5%)	0.159

*Data extrapolated from Matloub et al.³

^†Number of patients treated on the oral methotrexate regimens from the original CCG-1991.

SIG, significant.

Table 3 Demographics and clinical features of patients treated on CCG-1961 (regimen D) versus comparable regimen from the CCG-1961 clinical trial

	CCG-1961		P-value
	Current study	Seibel et al. study*
	CCG-1961 (regimen D)	CCG-1961 (equivalent regimen)
	n (%)	n (%)
Number of patients	72	650^†
Age (years)
1–9.9	51 (70.8)	229 (35.2)	0.001
10–15.9	21 (29.2)	334 (51.4)	0.001
Over 16	NA	87 (13.4)	NA
Sex
Female	30 (41.7)	284 (43.7)	0.839
Male	42 (58.3)	366 (56.3)
Ethnicity	Arabs/Middle Eastern	Heterogeneous
WBC count^‡
WBC <50 × 10^9/l	28 (38.9)	337 (51.8)	0.142
WBC (≥50 and <200) × 10^9/l	35 (48.6)	241 (37.1)	0.075
WBC ≥200 × 10^9/l	9(12.5)	69 (10.6)	0.773
Immunophenotyping^‡
B cell	59 (81.9)	449 (69.1)	PNC
T cell	13 (18.1)	112 (17.2)	PNC

*Data extrapolated from Siebel et al.⁴

^†Number of patients treated on augmented regimen (IPII) from the original CCG-1961.

^‡Percentages do not add up to 100%.

PNC, P-value not computed because of missing data; NA, not applicable.

Table 4 Comparison of outcomes of patients treated on CCG-1961 (regimen D) versus comparable regimen from the CCG-1961 clinical trial

	CCG-1961		P-value
	Current study	Seibel et al. study*
	CCG-1961 (regimen D)	CCG-1961 (equivalent regimen)
Number of patients (RER)	72	650^†
Study design	Retrospective	Prospective
	Single center	Multicenter
Study period	January 2001–December 2007	September 1996–May 2002
Five-year OS	85.8 ± 4.2%	88.7 ± 1.9%	NS
Five-year EFS	78.9 ± 4.8%	81.2 ± 2.4%	NS
Relapse/events: RER	7 (9.7%)	96 (14.8%)	0.325
Isolated BM relapse	1 (1.4%)	49 (7.5%)	0.050
Isolated CNS relapse	1 (1.4%)	29 (4.5%)	0.349
Combination	5 (6.9%)	15 (2.3%)	0.040
Testicular	0	2 (0.3%)	1.000
Other	0	1 (0.1%)	1.000
Death in remission	4 (5.6%)	12 (1.8%)	0.066

*Data extrapolated from Siebel et al.⁴

^†Number of patients treated on augmented regimen from the original CCG-1961.

NS, not significant.

Matloub Y, Bostrom BC, Hunger SP, Stork LC, Angiolillo A, Sather H, et al. Escalating intravenous methotrexate improves event-free survival in children with standard-risk acute lymphoblastic leukemia: a report from the children's oncology group. Blood. 2011;118:243–51. doi: 10.1182/blood-2010-12-322909

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Siebel NL, Steinherz PG, Sather HN, Nachman JB, Delaat C, Ettinger LJ, et al. Early postinduction intensification therapy improves survival for children and adolescents with high-risk acute lymphoblastic leukemia: a report from the children's oncology group. Blood. 2008;111:2548–55. doi: 10.1182/blood-2007-02-070342

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