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). To evaluate the safety profile and efficacy of TEM in this rare tumor entity, further data collection in an unselected routine clinical patient population is useful.
A German multicenter registry for rel/refr MCL pts treated with TEM was started in Germany in Oct 2009 with regulatory and ethic committeés approval. Objectives are the evaluation of the safety profile, 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 2017, 55 pts were recruited in 30 study sites. Baseline characteristics are available for 55 pts: 69.1% male; median age 74.4 years; bone marrow involvement in 38.2% of the pts; ECOG PS (n = 54) 0 or 1 in 83.3%, ECOG PS 2 in 16.7%. According to MIPI score (n = 53), 20.8%, 34.0%, and 45.2% are classified as low, intermediate, and high risk at the time of enrollment. Median number of prior therapies is 2 with 43.6% treated in ≥ 4th line. Most common drug-related toxicities of any grade (incidence ≥ 15%) are observed in following categories: blood/lymphatic system disorders (49.1%), gastrointestinal disorders (27.3%), general disorders (21.8%), and skin/subcutaneous tissue disorders (18.2%). Efficacy analyses are available for 39 assessable pts with an objective response in 30.8%, a clinical benefit (CR, PR, MR and SD) in 59.0% and PD in 41.0% of the pts. Median PFS for all pts is 3.6 mo. For the subgroup of pts treated with TEM in 2nd and 3rd line PFS is 3.3 mo, for ≥ 4th line pts 4.9 mo.
The registry was started to evaluate the safety and efficacy of TEM in pts with rel/refr MCL in routine clinical practice. In this comparatively 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. Dreyling, G. Hess: Consultant Pfizer Pharma GmbH, Honoraria from Pfizer GmbH. G. Krekeler: Employee of Pfizer Pharma GmbH.