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NON-INTERVENTIONAL RETROSPECTIVE MULTICENTER STUDY EVALUATING REAL WORD IDELALISIB USE IN CLL AND INHL PATIENTS ENROLLED IN THE FRENCH EARLY ACCESS PROGRAM (EAP)

PB1893

Ysebaert, L.1; Feugier, P.2; Salles, G.3; Durot, E.4; Talbot, A.5; Choquet, S.6; Kergaravat, Glorian D.7; Simpson, A.8; Ramroth, H.9; Abdelhadi, T.10; Haioun, C.11; Troussard, X.12

doi: 10.1097/01.HS9.0000566076.08559.69
Publication Only: Chronic lymphocytic leukemia and related disorders - Clinical
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1Hematology, IUCT, Toulouse

2Hematology, CHU Nancy, Vandoeuvre Les Nancy

3Hematology, Hospices Civils de Lyon - Université de Lyon, Pierre-Bénite

4Hematology, CHU de Reims, Hopital Robert Debré, Reims

5Immunohematology, Hopital Saint Louis

6Hematology, Hopital Pitié Salpêtrière, Paris

7Clinical Operation, Gilead Sciences, Boulogne Billancourt, France

8Clinical Datamanagment

9Pharmacovigilance & Epidemiology, Gilead Sciences, Uxbridge, United Kingdom

10Medical Affairs, Gilead Sciences, Boulogne Billancourt

11Lymphoid Malignacies Unit, Hopital Henri Mondor, Créteil

12Hematology, C.H.U. de Caen, Caen, France

Please indicate where the abstract has been published before: ASH Abstract-Blood 2018 132:5924.

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Background:

Idelalisib (IDELA), a PI3K-delta inhibitor, is indicated for the treatment of adult patients (pts) with chronic lymphocytic leukemia (CLL) and for pts with follicular lymphoma refractory to two prior lines of treatment. Its clinical efficacy and safety profile have been established in clinical trials, however, there is a paucity of real-word data regarding its use. This study was initiated to describe pts who started IDELA treatment during the French EAP.

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Aims:

The aims of this retrospective multicenter study were to describe the effectiveness, adverse events (AE), adverse drug reaction (ADR), serious ADR (SADR), AE of special interest (AESI: diarrhea/colitis, pneumonitis, liver enzyme elevation, neutropenia, infection, and rash), and IDELA use in adult pts previously enrolled in the EAP between June and October 2014 for CLL or iNHL.

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Methods:

Physicians who enrolled at least one patient in the EAP were contacted to participate in this study. For each patient, 12 months follow up data were collected and monitored by the Lymphoma Academic Research Organization (LYSARC). The primary endpoint was the overall response rate (ORR) at 6 months. Statistical analysis was descriptive

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Results:

Seventy-five pts were included, 41 in CLL group (39 in third-line or later line and 2 in first-line with del(17p)/TP53m unsuitable for chemo-immunotherapy and 34 in iNHL group, all refractory after two prior lines of treatment (table 1). In CLL/iNHL groups respectively, median IDELA treatment duration was 25.1 weeks/18.9 weeks, ORR was 82.8%/56.5% at 6 months (data reported in 29 pts/23 pts), median PFS was not reached (NR)/ 6.7 months, median time to next treatment (TTNT) defined as the time between the date of the last IDELA intake and the start of next treatment after progression of disease (PD) was NR/11.6 months, median OS was NR in both groups and OS estimate at 12 months was 69.9%/67.6% (table 2). In CLL/iNHL groups respectively, at least one ADR related to IDELA was reported during the study in 35 pts (85.4%)/26 pts (76.5%) and SADR related to IDELA in 23 pts (56.1%)/15 pts (44.1%), most frequently reported AEs were: infections and infestations 61.0%/50.0% [Pneumocystis jirovecii pneumonia (PJP) 2.4%/5.9%], gastrointestinal disorders 56.1%/35.3% (diarrhea 41.5%/29.4%), blood and lymphatic system disorders 41.5%/41.2% (neutropenia 17.1%/23.5%) and lab investigation 43.9%/47.1% (hepatic enzyme increase 19.5%/14.7%). 24 pts (58.5%)/15 pts (44.1%) interrupted IDELA temporarily for AE, 6 pts (14.6%)/8 pts (23.5%) discontinued permanently for AE, 38 pts (92.7%)/25 pts (73.5%) had at least one AESI and 3 pts (7.3%)/6 pts (17.6%) permanently discontinued IDELA for AESI. Deaths were reported during the study in 13 pts (31.7%)/11 pts (32.4%), due to PD (46.2%/72.7%).

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Figure

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Summary/Conclusion:

The results of this study of heavily pretreated CLL and iNHL population indicate that IDELA is an effective treatment in routine clinical practice with an acceptable safety profile. ORR at 6 months was 83%/56.5% in CLL/iNHL mirroring those of clinical trials (Furman NEJM 2014, Gopal NEJM 2014). The pattern of AEs corresponds to that reported in previous clinical studies and might be improved by specific management of AESI.

Copyright © 2019 The Authors. Published by Wolters Kluwer Health Inc., on behalf of the European Hematology Association.