Temsirolimus (TEM), an mTOR inhibitor, is approved in the EU for the treatment of pts with rel/refr MCL. A pivotal study demonstrated significantly longer progression free survival (PFS) with TEM (175 mg weekly for 3 weeks followed by 75 mg weekly) in rel/refr MCL pts compared to investigatoŕs choice therapy (4.8 vs 1.9 months (mo); P = .0009, HR 0.04). To evaluate safety and efficacy of TEM in this rare tumor entity, further collection of data in a post-approval prospective non-interventional trial is useful.
A German multicenter registry for rel/refr MCL pts treated with TEM was started in Germany in Oct 2009 (NCT00700258). Objectives are the evaluation of safety, tolerability and anti-tumor activity of TEM as well as patient's profile including comorbidities, characteristics, and the sequence of systemic therapies.
From Oct 2009 to Feb 2018, 56 pts were recruited in 31 study sites. Baseline characteristics are available for 56 pts: 69.6% male; median age 74.4 years; ECOG PS 0 or 1 in 83.6%, PS 2 in 16.4%. According to MIPI score (n = 54), 20.4%, 33.3%, and 46.3% are classified as low, intermediate, and high risk at the time of enrollment. Median number of prior therapies is 2 with 44.7% treated in ≥ 4th line. Drug related adverse and serious adverse events are observed in 67.9% and 12.5% of pts, respectively. Most common drug-related toxicities of any grade (incidence ≥ 15%) in the following categories: blood/lymphatic system disorders (48.2%), gastrointestinal disorders (28.6%), general disorders (23.2%), and skin/subcutaneous tissue disorders (17.9%). Efficacy analyses are available for 40 assessable pts with an objective response rate (ORR) of 30%, a clinical benefit rate of 60% and PD in 40% of the pts. Median PFS for all pts is 3.7 mo and for pts who achieved an OR with TEM 6.3 mo. Median OS for all pts is 14.8 mo.
The registry was started to evaluate the safety and efficacy of TEM in pts with rel/refr MCL in routine clinical practice. 46.3% high-risk pts were included in the analysis. In this poor-prognosis patient population, TEM showed a predictable, manageable tolerability profile. Efficacy parameters were consistent with published phase III data.
Clinical trial identification
Legal entity responsible for the study
Pfizer Pharma GmbH.
Pfizer Pharma GmbH.
M. Woike, G. Krekeler: Employee: Pfizer Pharma GmbH. All other authors have declared no conflicts of interest.