A phase 1 study of inotuzumab ozogamicin in pediatric relapsed/refractory acute lymphoblastic leukemia (ITCC-059 study).


Journal

Blood
ISSN: 1528-0020
Titre abrégé: Blood
Pays: United States
ID NLM: 7603509

Informations de publication

Date de publication:
25 03 2021
Historique:
received: 26 06 2020
accepted: 29 09 2020
pubmed: 18 10 2020
medline: 24 8 2021
entrez: 17 10 2020
Statut: ppublish

Résumé

This phase 1 study investigated the recommended phase 2 dose (RP2D) of inotuzumab ozogamicin (InO), a CD22-directed antibody-drug conjugate, in pediatric patients with multiple relapsed/refractory (R/R) CD22+ acute lymphoblastic leukemia (ALL). Patients (age ≥1 year or <18 years) received 3 doses of InO (days 1, 8, and 15) per course. Dose escalation was based on dose-limiting toxicities (DLTs) during course 1. Dose level 1 (DL1) was 1.4 mg/m2 (0.6, 0.4, 0.4 mg/m2) and DL2 was 1.8 mg/m2 (0.8, 0.5, 0.5 mg/m2). Secondary end points included safety, antileukemic activity, and pharmacokinetics. Twenty-five patients (23 evaluable for DLTs) were enrolled. In course 1, the first cohort had 1 of 6 (DL1) and 2 of 5 (DL2) patients who experienced DLTs; subsequent review considered DL2 DLTs to be non-dose-limiting. Dose was de-escalated to DL1 while awaiting protocol amendment to re-evaluate DL2 in a second cohort, in which 0 of 6 (DL1) and 1 of 6 (DL2) patients had a DLT. Twenty-three patients experienced grade 3 to 4 adverse events; hepatic sinusoidal obstruction syndrome was reported in 2 patients after subsequent chemotherapy. Overall response rate after course 1 was 80% (95% confidence interval [CI], 59% to 93%) (20 of 25 patients; DL1: 75% [95% CI, 43% to 95%], DL2: 85% [95% CI, 55% to 98%]). Of the responders, 84% (95% CI, 60% to 97%) achieved minimal residual disease (MRD)-negative complete response, and 12-month overall survival was 40% (95% CI, 25% to 66%). Nine patients received hematopoietic stem cell transplantation or chimeric antigen receptor T cells after InO. InO median maximum concentrations were comparable to simulated adult concentrations. InO was well tolerated, demonstrating antileukemic activity in heavily pretreated children with CD22+ R/R ALL. RP2D was established as 1.8 mg/m2 per course, as in adults. This trial was registered at https://www.clinicaltrialsregister.eu as EUDRA-CT 2016-000227-71.

Identifiants

pubmed: 33067614
pii: S0006-4971(21)00665-0
doi: 10.1182/blood.2020007848
pmc: PMC7995290
doi:

Substances chimiques

Antineoplastic Agents, Immunological 0
Inotuzumab Ozogamicin P93RUU11P7

Types de publication

Clinical Trial, Phase I Journal Article Multicenter Study Research Support, Non-U.S. Gov't

Langues

eng

Sous-ensembles de citation

IM

Pagination

1582-1590

Commentaires et corrections

Type : CommentIn

Informations de copyright

© 2021 by The American Society of Hematology.

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Auteurs

Erica Brivio (E)

Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands.
Department of Pediatric Oncology, Erasmus Medical Center (Erasmus MC) Sophia Children's Hospital, Rotterdam, The Netherlands.

Franco Locatelli (F)

Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, Ospedale Pediatrico Bambino Gesù, Sapienza University of Rome, Rome, Italy.

Marta Lopez-Yurda (M)

Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands.
Netherlands Cancer Institute, Amsterdam, The Netherlands.

Andrea Malone (A)

Children's Health Ireland at Crumlin, Dublin, Ireland.

Cristina Díaz-de-Heredia (C)

Department of Pediatric Hematology and Oncology, University Hospital Vall d'Hebron, Barcelona, Spain.

Bella Bielorai (B)

Department of Pediatric Hematology-Oncology, The Edmond and Lily Safra Children's Hospital, Sheba Medical Center, Ramat-Gan, Israel.

Claudia Rossig (C)

Department of Pediatric Hematology and Oncology, University Children's Hospital Münster, Germany.

Vincent H J van der Velden (VHJ)

Department of Immunology, Erasmus MC, University Medical Center Rotterdam, Rotterdam, The Netherlands.

Anneke C J Ammerlaan (ACJ)

Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands.

Adriana Thano (A)

Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands.
Netherlands Cancer Institute, Amsterdam, The Netherlands.

Inge M van der Sluis (IM)

Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands.
Department of Pediatric Oncology, Erasmus Medical Center (Erasmus MC) Sophia Children's Hospital, Rotterdam, The Netherlands.

Monique L den Boer (ML)

Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands.
Department of Pediatric Oncology, Erasmus Medical Center (Erasmus MC) Sophia Children's Hospital, Rotterdam, The Netherlands.
The Oncode Institute, Utrecht, The Netherlands.

Ying Chen (Y)

Pfizer Global Product Development, San Diego, CA.

Barbara Sleight (B)

Pfizer Inc, Groton, CT.

Benoit Brethon (B)

Department of Pediatric Hematology, Hôpital Robert-Debré, Assistance Publique-Hôpitaux de Paris, Paris, France.

Karsten Nysom (K)

Department of Paediatrics and Adolescent Medicine, Juliane Marie Centre, Rigshospitalet, Copenhagen, Denmark.

Lucie Sramkova (L)

Department of Paediatric Haematology and Oncology, 2nd Faculty of Medicine, Charles University in Prague and Motol University Hospital, Prague, Czech Republic.

Ingrid Øra (I)

Department of Pediatric Oncology and Hematology, Childhood Cancer Research Unit, Lund University Hospital, Lund, Sweden.
Pediatric Oncology and Hematology, Karolinska Hospital, Stockholm, Sweden.

Luciana Vinti (L)

Department of Pediatric Hematology/Oncology and Cell and Gene Therapy, Ospedale Pediatrico Bambino Gesù, Sapienza University of Rome, Rome, Italy.

Christiane Chen-Santel (C)

Department of Pediatrics, Division of Oncology and Hematology, Charité-Universitätsmedizin Berlin, Berlin, Germany; and.
Universitätsmedizin Rostock, Kinder- und Jugendklinik, Rostock, Germany.

Christian Michel Zwaan (CM)

Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands.
Department of Pediatric Oncology, Erasmus Medical Center (Erasmus MC) Sophia Children's Hospital, Rotterdam, The Netherlands.

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