Synopsis of results of case-control studies comparing effect of ruxolitinib versus conventional therapy on survival in myelofibrosis
| Reference . | Case identification . | Method of matching . | Cohort, n . | Time point for survival measurement . | Survival . | Difference in survival . | Conclusion reported . | ||
|---|---|---|---|---|---|---|---|---|---|
| Control . | Ruxolitinib . | Control cohort . | Ruxolitinib cohort . | ||||||
| 10 | Ruxolitinib and controls: patients at Mayo Clinic in most recent 10-y period | DIPSS-plus score | 410 | 51 | Ruxolitinib: therapy initiation Controls: initial referral to center | NR | DIPPS-plus low, 185 mo Intermediate-1, 78 mo Intermediate-2, 35 mo High risk, 16 mo | P = .58 (with adjustment for DIPSS-plus) | No significant difference in survival rate for ruxolitinib-treated patients compared with those treated with standard therapy |
| 11 | Ruxolitinib: patients at MDACC who participated in INCB 18424-251 trial Controls: historical control group* | Trial enrollment criteria | 310 | 107 | Ruxolitinib and controls: first observation at center | NR | OS rate, 69% | HR, 0.58 (95% CI, 0.39-0.85); P = .005 (with adjustment for IPSS risk status) | Seems to be survival advantage for patients treated with ruxolitinib (finding should be interpreted with caution) |
| 12 | Ruxolitinib: patients who received ruxolitinib in randomized treatment arm or after crossover from best available therapy in COMFORT-II trial Controls: patients entered in database and not receiving any experimental drug | IPSS intermediate-2 or high risk Blasts <10% | 350 | 100 | Ruxolitinib: therapy initiation Controls: first documentation of intermediate-2– or high-risk status | Median OS, 3.5 y (95% CI, 3.0-3.9) | Median OS, 5 y (95% CI, 2,9-7.8) | HR, 0.61 (95% CI, 0.4-0.9); P = .005 | Risk of death might be reduced by 40% to 50% by introducing ruxolitinib in treatment of patients with primary myelofibrosis |
| 13 | Momelotinib: patients who received momelotinib in a phase 1/2 clinical trial (NCT00935987) Controls: retrospective cohort of JAK inhibitor treatment–naive patients from authors’ institutional database | DIPSS-plus risk status | 442 | 100 | Momelotinib: from tdate of study entry Controls: NR | Median OS, 3.2 y | Median OS, 3 y | P = .44 | Comparison of momelotinib-treated patients with risk-matched myelofibrosis cohort not receiving treatment with momelotinib did not reveal significant difference in survival data |
| 14 | Ruxolitinib and controls: SEER (ICD-O-3) | By y of diagnosis | 975 | 1045 | Diagnosis | 4-y RSR, 54% | 4-y RSR, 57% | P = .776 | No difference in survival between pre- and postruxolitinib cohorts |
| 1 | Ruxolitinib and controls: ad hoc registry (ERNEST study) | Propensity score matching analysis on baseline covariates including DIPSS | 50 | 50 | Start of hydroxyurea or ruxolitinib therapy | Median OS, 3.4 y | Median OS, 7.7 y | P = .002 | Compared with treatment with hydroxyurea, ruxolitinib treatment was associated with significant OS benefit |
| Reference . | Case identification . | Method of matching . | Cohort, n . | Time point for survival measurement . | Survival . | Difference in survival . | Conclusion reported . | ||
|---|---|---|---|---|---|---|---|---|---|
| Control . | Ruxolitinib . | Control cohort . | Ruxolitinib cohort . | ||||||
| 10 | Ruxolitinib and controls: patients at Mayo Clinic in most recent 10-y period | DIPSS-plus score | 410 | 51 | Ruxolitinib: therapy initiation Controls: initial referral to center | NR | DIPPS-plus low, 185 mo Intermediate-1, 78 mo Intermediate-2, 35 mo High risk, 16 mo | P = .58 (with adjustment for DIPSS-plus) | No significant difference in survival rate for ruxolitinib-treated patients compared with those treated with standard therapy |
| 11 | Ruxolitinib: patients at MDACC who participated in INCB 18424-251 trial Controls: historical control group* | Trial enrollment criteria | 310 | 107 | Ruxolitinib and controls: first observation at center | NR | OS rate, 69% | HR, 0.58 (95% CI, 0.39-0.85); P = .005 (with adjustment for IPSS risk status) | Seems to be survival advantage for patients treated with ruxolitinib (finding should be interpreted with caution) |
| 12 | Ruxolitinib: patients who received ruxolitinib in randomized treatment arm or after crossover from best available therapy in COMFORT-II trial Controls: patients entered in database and not receiving any experimental drug | IPSS intermediate-2 or high risk Blasts <10% | 350 | 100 | Ruxolitinib: therapy initiation Controls: first documentation of intermediate-2– or high-risk status | Median OS, 3.5 y (95% CI, 3.0-3.9) | Median OS, 5 y (95% CI, 2,9-7.8) | HR, 0.61 (95% CI, 0.4-0.9); P = .005 | Risk of death might be reduced by 40% to 50% by introducing ruxolitinib in treatment of patients with primary myelofibrosis |
| 13 | Momelotinib: patients who received momelotinib in a phase 1/2 clinical trial (NCT00935987) Controls: retrospective cohort of JAK inhibitor treatment–naive patients from authors’ institutional database | DIPSS-plus risk status | 442 | 100 | Momelotinib: from tdate of study entry Controls: NR | Median OS, 3.2 y | Median OS, 3 y | P = .44 | Comparison of momelotinib-treated patients with risk-matched myelofibrosis cohort not receiving treatment with momelotinib did not reveal significant difference in survival data |
| 14 | Ruxolitinib and controls: SEER (ICD-O-3) | By y of diagnosis | 975 | 1045 | Diagnosis | 4-y RSR, 54% | 4-y RSR, 57% | P = .776 | No difference in survival between pre- and postruxolitinib cohorts |
| 1 | Ruxolitinib and controls: ad hoc registry (ERNEST study) | Propensity score matching analysis on baseline covariates including DIPSS | 50 | 50 | Start of hydroxyurea or ruxolitinib therapy | Median OS, 3.4 y | Median OS, 7.7 y | P = .002 | Compared with treatment with hydroxyurea, ruxolitinib treatment was associated with significant OS benefit |
CI, confidence interval; DIPSS, Dynamic International Prognostic Scoring System; HR, hazard ratio; ICD-O-3, International Classification of Diseases for Oncology, third edition; IPSS, International Prognostic Scoring System; MDACC, MD Anderson Cancer Center; NR, not reported; OS, overall survival; RSR, relative survival rate; SEER, Surveillance, Epidemiology, and End Results.
Databases of 3 international institutions.