969PEVALUATION OF SAFETY, TOLERABILITY AND EFFICACY OF TEMSIROLIMUS IN PATIENTS WITH RELAPSED OR REFRACTORY MANTLE CELL LYMPHOMA (REL/REFR MCL) IN ROUTINE CLINICAL PRACTICE

ABSTRACT Aim: Temsirolimus (TEMS), an mTOR-inhibitor, is approved in the EU for the treatment of patients (pts) with relapsed or refractory (rel/refr) MCL. A pivotal study demonstrated significantly longer progression free survival with TEMS (175 mg weekly for 3 weeks followed by 75 mg weekly) in rel/refr MCL pts compared to investigatoŕs choice therapy (4.8 mo vs 1.9 mo; P = .0009). To evaluate safety and efficacy of TEMS in an unselected patient population during clinical routine, a prospective non-interventional study with TEMS in rel/refr MCL pts is useful. Here we report on interim results of the study. Methods: A German multicenter registry for rel/refr MCL pts treated with TEMS was started in Oct 2009 (NCT00700258). Objectives are evaluation of the safety profile of TEMS, tolerability and anti-tumor activity of TEMS, patientś profile and the sequence of systemic therapies. Results: From Oct 2012 to Feb 2014, 21 study sites recruited 38 pts. Baseline characteristics are available for 38 pts: 73.7% male; median age 73.6 yrs; ECOG PS 0 or 1 in 81.1%, ECOG PS 2 in 18.9% of the pts; MIPI score: 25.0%, 30.6%, and 44.4% of the pts are classified as low, intermediate and high risk at the time of enrollment, bone marrow involvement: 44.7% of the pts. Median time between diagnosis and start oft treatment with TEMS is 3.4 yrs (range 0.4 - 14.9). The median number of prior therapies is 2.5 (range 1 - 9) with 50% of the pts treated in ≥4th line. Most common adverse events (≥ 15%) are thrombocytopenia (39.5%), anemia (15.8%) and general physical health deterioration (18.4%). Severe adverse events (drug related) are general, metabolic, psychiatric, skin, renal, gastrointestinal and blood system disorders (in 1 pt each) and infections (in 2 pts). Preliminary efficacy analyses are available for 27 assessable pts and show an objective response in 8 pts (1CR and 7 PR, 29.6%), a clinical benefit (CR, PR, MR and SD) in 16 pts (59.3%) and PD in 11 pts (40.7%). Median PFS is 4.2 months. Conclusions: The registry was started to evaluate the safety and efficacy of TEMS in pts with rez/refr MCL in the routine clinical practice. In this here included patient collective with 44.4% high-risk pts, TEMS shows a predictable, manageable tolerability profile and efficacy remains comparable with phase III data. Disclosure: G. Krekeler: Employee of Pfizer GmbH, Germany; M.H. Dreyling: membership on an advisory board; G. Hess: membership on an advisory board, cooperate-sponsored research; D. Kalanovic: Employee of Pfizer GmbH.