Exclusion from Acute Myeloid Leukemia (AML) Clinical Trials of Patients Unlikely To Respond.

Introduction: Patients with newly diagnosed AML are increasingly being enrolled on industry-sponsored clinical trials. These trials routinely exclude patients with Zubrod performance status (PS) >2, bilirubin >1.9 mg/dL, or creatinine >1.9 mg/dL; such patients are well-known to be relatively unlikely to respond (UTR). Here we quantify the effect of this practice on inclusion of UTR patients in clinical trials.

Patients and Methods: Overall, 2323 adults with newly-diagnosed AML (no acute promyelocytic leukemia) were registered on clinical trials (industry-sponsored and other) from 1991–2006. Considering patients as UTR if they fulfilled any of the criteria noted above, we examined rates of enrollment of UTR patients over time:

Results: Thus, while more patients are being enrolled on clinical trials, fewer UTR patients are being enrolled particularly over the last 5 years (p = .04) with a concomitant decrease in the proportion of such patients enrolled (p<.0001 over the 16 years in question). Findings are qualitatively the same in older patients.

Conclusion: Newer clinical trials may be less applicable to UTR patients than previous trials. There is a need for clinical trials specific to the UTR population, the group most in need of novel therapies.