TABLE IV.

Quality of the studies

Reference	Study design	Outcomes		Follow-up duration	Not included in final analysis
		Primary	Secondary		(n)	(%)
Randomized studies
Ottmann et al., 200710 Wassmann et al., 200311	Randomized controlled trial	Hematologic response after induction	Complete molecular response Recurrence Death Discontinuation of imatinib Toxicity	Median: 11 months	2/55	3
Lilly et al., 201012	Randomized open-label phase iii	Major hematologic response	Overall response Cytogenetic response Progression-free survival Overall survival Safety Time to response	2 Years	1
Observational studies
Ottmann et al., 200213	Prospective case series	Complete hematologic response Marrow complete response Partial response	Cytogenic response Time to progression Overall survival	Approximately 12 months	Not stated
Wassmann et al., 200214	Case series	Efficacy Tolerability	Response to hematopoietic stem-cell transplantation	Median: 13 months	8/30 (not transplanted)
Pfeifer et al., 200315	Retrospective case series	Incidence and risk factors for cns relapse	Complete remission	Not stated	0
Scheuring et al., 200316	Retrospective case series	Predictors Prognosis of BCR-ABL to duration of response	Complete remission Molecular response Time to progression Overall survival	4 Weeks	Variable
Shimoni et al., 200317	Case series	Stem-cell transplantation and donor lymphocyte infusion outcomes	Complete remission Cytogenic response Toxicity Graft-vs-host disease Engraftment	Median: 10 months	0
Wassmann et al., 200318	Prospective case series	Efficacy Tolerability Safety	Time to progression Relapse Refractoriness Overall survival	Median: 16 months	0
Houot et al., 200419	Retrospective case series	Characteristics Response	Not stated	Median: 8 months	0
Piccaluga et al., 200420	Case series	Significance of molecular remission	Not stated	Up to 40 months	0
Thomas et al., 200421	Prospective cohort	Not stated	Not stated	Median: 20 months	0
Towatari et al., 200422	Prospective case series	Complete remission	Toxicity Response duration Survival	Median: 12 months	0
Wassmann et al., 200423	Retrospective case series	Factors predicting response and response duration	Time to progression Overall survival	Not stated	0
Brandwein et al., 200524	Retrospective case series	Predictors of response	Complete remission Progression-free survival Overall survival	Not stated	0
Lee et al., 200525	Prospective cohort	Complete remission Remission duration Overall survival	Not stated	Median: 26 months	0
Lee et al., 200526 Lee et al., 200327	Prospective cohort	Time to stem-cell transplantation Transplantation outcome	Response rate Overall survival Disease-free survival	Median: 25 months	0
Potenza et al., 200528	Case series	Efficacy	Minimal residual disease	24 Months	0
Deininger et al., 200629	Retrospective cohort	Overall survival Progression-free survival Relapse rate Time to engraftment	Not stated	21.6 Months	0
Delannoy et al., 200630	Prospective cohort	Overall survival	Complete remission Relapse-free survival Toxicity Minimum residual disease	Median: 24 months survivors 14	1/30 (complete response)	3
Kantarjian et al., 200631	Prospective case series	Safety Hematologic response Cytogenic response Tolerability	Not stated	12 Months	Not stated; 45% did not complete the study
Rea et al., 200632	Case series	Complete hematologic response	Overall survival Disease-free survival	Median: approximately 8 months	0
Wassmann et al., 200633	Prospective cohort	Feasibility Toxicity	Complete remission Response rate Transplantation rate Minimal residual disease	Not stated, to stem-cell transplantation	0
Yanada et al., 200634, 200835; Zembutsu et al., 200736	Prospective cohort	Complete remission	Response duration Overall survival Toxicity	38 Months	0
Carpenter et al., 200737	Prospective case series	Safety and tolerability 90 days after stem-cell transplantation	Survival Molecular relapse	Median: 11 months	0
de Labarthe et al., 200738	Prospective case series	Not stated	Not stated	Median: 18 months	0
Ottmann et al., 200739	Prospective case series	Major hematologic response plus overall hematologic response	Cytogenic response Duration of hematologic response Safety Tolerability	Minimum: 8 months	0
Pfeifer et al., 200740	Retrospective	Kinase domain mutation	Kinase domain mutation and hematologic response Relapse	na	7/55	12
Vignetti et al., 200741	Prospective case series	Complete hematologic response Complete molecular response Complete remission Toxicities	Not stated	Maximum: 32 months	1/30	3
Burke et al., 200942	Prospective case series	Overall survival Relapse-free survival Risk of relapse Cardiac toxicity	Not stated	Median: 0.93 years	0
Nicolini et al., 200943	Cross-sectional survey	Mutation testing Overall survival Progression-free survival	Not stated	Not stated	0
Sakamaki et al., 200944	Prospective case series	Hematologic response Major cytogenic response Safety	Not stated	Not stated	1
Tojo et al., 200945	Prospective case series	Hematologic response Complete remission Pharmacokinetics Safety	Not stated	Minimum: 12 months	0
Bassan et al., 201046 Bassan et al., 200947 (abstract)	Prospective case series	Overall survival	Disease-free survival Treatment-related mortality Cumulative incidence relapse Minimal residual disease	Median: 5 years	6
Li et al., 201048	Retrospective case series	Overall survival Disease-free survival Complete remission Molecular complete response Relapse	Not stated	Median: 12.5 months	0
Nishiwaki et al., 201049	Retrospective case series	Hematologic response Overall survival Minimal residual disease	Not stated	Maximum: 2 years	0
2Olsson–Stromberg et al., 201050	Not stated	Mutation analysis	Not stated	Not stated	0
Ravandi et al., 201051 Ravandi et al., 200852 (abstract)	Prospective case series	Minimal residual disease Complete remission Disease-free survival Event-free survival Overall survival	Adverse events	Median: 14 months	2/35 (complete response)	6
Ribera et al., 201053 Ribera et al., 200454 (abstract)	Prospective case series	Complete hematologic response Percentage of patients reaching transplantation Feasibility of imatinib treatment	Toxicity Safety Disease-free survival Overall survival	Median: 4.1 years	0
Riva et al., 201055	Prospective cohort	Minimal residual disease Hematologic response	Not stated	Minimum: 4 months	0
Chen et al., 201156	Prospective case series	Minimal residual disease Overall survival Disease-free survival Response rate Safety	Not stated	Median: 24 months	0
Foà et al., 201157 Foà et al., 200758 (abstract)	Prospective case series	Complete hematologic response	Toxicity Immunophenotypic response rate Molecular response Disease-free survival Response rate Overall survival	Median: 24.8 months	2/55	4
Mizuta et al., 201159 Yanada et al., 200634, 200835; Zembutsu et al., 200736	Prospective cohort	Transplantation outcome Overall survival	Disease-free survival Response duration	Not stated	0
Wang et al., 201160	Retrospective case series	Complete remission Overall survival Relapse-free survival	Not stated	Approximately 40 months	0
Bose et al., 201261	Prospective case series	Safety Complete hematologic response Pharmacodynamics	Not stated	Not stated	1/22 (cml)
Caocci et al., 201262	Prospective case series	Minimal residual disease	Not stated	17 Months after stem-cell transplantation	0
Chen et al., 201263	Prospective case series	Safety	Response rate Disease-free survival Overall survival	31 Months	2/82 (imatinib group)	2
Lee et al., 201264	Prospective case series	Complete hematologic response Major molecular response Complete molecular response Overall survival Disease-free survival	Not stated	61 Months	18
Pfeifer et al., 201265 Patients from Ottmann et al., 200739; Ottmann et al., 200213; Wassmann et al., 200423	Prospective case series	Mutation status	Not stated	Multiple	Not available
Thyagu et al., 201266	Prospective case series	Complete remission Major cytogenic response Overall survival Event-free survival	Not stated	85 Months	0
Abstracts
Dombret et al., 200467	Prospective case series	Increased molecular response Feasibility of autologous stem-cell collection	Safety	Not stated	Not stated
Thomas et al., 200468	Prospective case series	Not stated	Not stated	Median: 24 months	4/22	18
Wetzler et al., 200669	Case series	Not stated	Not stated	Not stated	11/18	65
Fielding et al., 200770	Prospective cohort	Role of etoposide or total body irradiation in sct	Not stated	More than 6 months	Not stated
Pasquini et al., 200771	Randomized controlled trial	Major hematologic response	Not stated	Median: 6.5 months	Not stated
Gambacorti–Passerini et al., 200872	Prospective case series	Complete hematologic response Major cytogenic response Adverse events	Not stated	Median: 6.1 weeks	0
Thomas et al., 200887	Prospective case series	Complete hematologic response Complete molecular response	Not stated	Not stated	Not stated
Arellano et al., 200973	Prospective case series	Complete hematologic response Complete molecular response Disease-free survival Overall survival	Not stated	Median: 18.3 months	1 (after complete cytogenic response)
Carella et al., 200974	Prospective case series	Complete remission Molecular response	Not stated	Not stated	Not stated
Pfeifer et al., 200975 (patients from Ottmann et al., 200739)	Prospective case series	Complete remission Minimal residual disease Mutational analysis	Disease-free survival Overall survival Response rate	Not stated	0
Ribera et al., 200454	Prospective case series	Complete remission Complete molecular response Major molecular response Disease-free survival Overall survival	Not stated	Median: 4.1 years	0
Pfeifer et al., 201076	Prospective case series	Complete remission Minimal residual disease Mutation analysis	Not stated	Median: 26 months	0
Rousselot et al., 201077	Prospective case series	Complete remission Relapse-free survival Cytogenetics	Not stated	Maximum: 4 years	0
Thomas et al., 201078	Prospective case series	Complete remission Major cytogenic response Overall survival	Not stated	Median: 16.3 months	0
Kim et al., 201179	Prospective case series	Hematologic complete response Major cytogenic response Overall survival Event-free survival Relapse-free survival	Not stated	Median: 77 months	0
Lee et al., 201180	Prospective case series	Complete remission Minimal residual disease Major molecular response	Not stated	Median: 1.4 months	0
Lee et al., 201181 Ravandi et al., 200852; Ravandi et al., 200982	Prospective case series	Complete remission Disease-free survival Overall survival	Not stated	Median: 10 months	6/36
Liu–Dumlao et al., 201183 Ravandi et al., 200982	Prospective case series	Complete remission Cytogenic response Molecular response	Not stated	Median: 26.1 months	Not stated
Pfeifer et al., 201184	Prospective case series	Minimal residual disease Remission duration Tolerability	Not stated	Median: 139 weeks	0
Brummendorf et al., 201285	Prospective case series	Major hematologic response Complete hematologic response	Not stated	Median: 30 months	Not stated
		Major cytogenic response Complete cytogenic response	Not stated	Median: 31 months	0
Cortes et al., 201286	Prospective case series	Adverse events Major hematologic response Molecular response Cytogenic response Dose-limiting toxicities	Not stated	Median: 56 weeks	0