Long-Term Clinical Outcomes of Tisagenlecleucel in Pediatric and Young Adult Patients With Relapsed/Refractory ALL
Abstract
We report the 5-year analysis of tisagenlecleucel in 79 pediatric and young adult patients with relapsed or refractory (r/r) B-cell ALL (B-ALL) from the global phase II ELIANA trial (ClinicalTrials.gov identifier: NCT02435849 ), with a median follow-up of 79.4 months. Tisagenlecleucel was administered as a single infusion with dosing normalized by weight in patients ≤50 kg. Key long-term end points included relapse-free survival (RFS), overall survival (OS), and safety. Censoring included loss to follow-up, withdrawal of consent, new anticancer therapy (± stem cell transplantation [SCT]), and death. The estimated 5-year RFS among responders (n = 70) with and without inclusion of SCT in censoring for new anticancer therapies was 47.3% and 51.0%, respectively. The median time to B-cell recovery was not reached with censoring for all further anticancer therapies, including SCT. The median OS was not reached. The estimated OS at 5 years was 55.0% and 62.4% with and without inclusion of SCT in censoring for new anticancer therapies, respectively. In total, 17 responders received a postinfusion SCT, 14 while still in complete remission. No new or unexpected adverse events were reported. These findings continue to support the potential of tisagenlecleucel as definitive therapy for many heavily pretreated pediatric and young adult patients with r/r B-ALL.
Article Details
Journal Info
Journal of Clinical Oncology
Lippincott Williams & Wilkins
Authors (29)
Stephan A. Grupp
Shannon L. Maude
Susana Rives
12Institut de Recerca Sant Joan de Déu, Barcelona, Spain
Hidefumi Hiramatsu
1Department of Pediatrics, Graduate School of Medicine, Kyoto University, Kyoto, Japan
Andre Baruchel
Peter Bader
3Division for Stem Cell Transplantation, Immunology, Department of Pediatrics, Goethe University, University Hospital, Frankfurt am Main, Germany
Henrique Bittencourt
7Pediatric Hematology-Oncology Division, Saint-Justine University Hospital Centre, Montreal, QC, Canada
Jochen Buechner
1Department of Pediatric Hematology and Oncology, Oslo University Hospital, Oslo, Norway
Theodore W. Laetsch
Division of Oncology, Department of Pediatrics, Children's Hospital of Philadelphia, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA
Barbara De Moerloose
Muna Qayed
Heather E. Stefanski
Center for International Blood and Marrow Transplant Research, NMDP, Minneapolis, MN
Kara L. Davis
Timothy A. Driscoll
Pediatric Transplant and Cellular Therapy, Duke University Medical Center, Durham, NC
Eneida Nemecek
2Oregon Health & Science University, Division of Hematology & Oncology, Knight Cancer Institute, Portland, United States
Christina Peters
2St. Anna Children's Cancer Research Institute, Vienna, Austria
Gregory Yanik
10University of Michigan, Ann Arbor, United States
Adriana Balduzzi
23Pediatric Hematopoietic Transplant Unit, Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy
Nicolas Boissel
Seong Lin Khaw
21Royal Children's Hospital and Murdoch Children's Research Institute, Children's Cancer Centre, Parkville, Australia
Joerg Krueger
6The Hospital for Sick Children, Toronto, Canada
John E. Levine
Icahn School of Medicine at Mount Sinai
Gary Douglas Myers
Division of Hematology/Oncology/Bone Marrow Transplant, Children's Mercy Kansas City, Kansas City, MO
Ranjan Tiwari
Novartis Healthcare Private Limited, Hyderabad, India
Darragh O'Donovan
33Novartis Pharmaceuticals Corporation, Dublin, Ireland
Rakesh Awasthi
32Novartis Pharmaceuticals Corporation, East Hanover, United States
Roberto Ramos
32Novartis Pharmaceuticals Corporation, East Hanover, United States
Jennifer Willert
Novartis Pharmaceuticals Corporation, San Franscisco, CA
Michael A. Pulsipher