The advantages we are enjoying these days of longer lives and better quality of life is largely the result of clinical trials, where the types of patients accepted and the care they receive is tightly controlled and often optimized to provide the best possible outcomes.
Once drugs are approved however, how they are used is left to the discretion of the treating physician, with varying influences from the manufacturer’s label prescribing approved usage, the patient’s preferences, appropriate guidelines, and increasingly, what insurance will cover.
Doctors can prescribe drugs off label for indications where there is good reason and data to indicate they will work. Again, insurance issues can block this from happening.
Doctors can also adjust the dosing and suggested monitoring schedule.
This “real world” use of medications is important in understanding our CLL treatment because efficacy often drops and complications may climb when drugs leave the tightly regulated realm of clinical trials and enter the “real world.”
Dr. Anthony Mato, who has recently moved from University of Pennsylvania to Memorial Sloan Kettering Cancer Center or MSKCC (contact information: Director, CLL Program, Memorial Sloan Kettering Cancer Center, 1275 York Ave, New York, NY 10065; Office number: 212-639-8596), has done important research in how chronic lymphocytic leukemia is treated in the community or “real-world”.
This is especially critical for a drug such as venetoclax where there are significant risks if the drug is not properly prescribed and monitored, and significant benefits when it is used appropriately to treat CLL.
I interviewed Dr. Mato at ASH 2017 (American Society of Hematology Annual Meeting and Exposition) in December just as he was making the transition from U. Penn to MSKCC) about his work as the lead researcher on the real-world data concerning the use of venetoclax.
Key Take Aways:
- 204 patients with chronic lymphocytic leukemia from 20 centers around the world were studied.
- Most patients tolerated the treatment well and side effects rarely led to discontinuation.
- The top three reasons for discontinuation were CLL progression (47%), Richter’s transformation (21%), and venetoclax toxicity (11%), most commonly low blood counts.
- Tumor lysis syndrome (TLS) was reassuringly rare among CLL patients being treated with Venetoclax in the community setting. Please see this article on how to properly take venetoclax and more on TLS and other complications.
- Risk factors for progression on venetoclax included prior use of drugs, such as ibrutinib or prior cellular therapy such as CAR-T or having the well-known poor prognostic markers, 17p deletion or a complex karyotype.
- The few CLL patients who fail venetoclax but had not had prior ibrutinib, may still respond to ibrutinib.
You can watch my interview with Dr. Mato below or read the transcript here.
Here is a link to the abstract with more numbers and details.
Patients worry that research results may get “lost in translation” when applied in the real world. This research is reassuring about the safety and efficacy of venetoclax in treating CLL in the community setting.
Brian Koffman, MD 1/30/18