Efficacy of anti PD-1 therapy in children and adolescent melanoma patients (MELCAYA study).

Adolescent Anti PD-1 Child Melanoma Outcome Safety

Journal

European journal of cancer (Oxford, England : 1990)
ISSN: 1879-0852
Titre abrégé: Eur J Cancer
Pays: England
ID NLM: 9005373

Informations de publication

Date de publication:
03 Sep 2024
Historique:
received: 26 08 2024
accepted: 27 08 2024
medline: 6 9 2024
pubmed: 6 9 2024
entrez: 5 9 2024
Statut: aheadofprint

Résumé

Data on the efficacy and safety of anti PD-1 antibodies in children and adolescents (CA) with melanoma are lacking. The aim of this study was to determine outcomes of CA melanoma patients receiving anti PD-1 antibodies. Melanoma patients ≤18 years treated with anti PD-1 were retrospectively retrieved from 15 academic centers. Information on histopathological diagnosis, surgical treatment, systemic therapy, objective response rate (ORR), safety profile was collected. Progression-free survival (PFS) and overall survival (OS) were assessed by Kaplan-Meier method. Between April 2016 and March 2024, 99 patients treated with systemic therapy were retrieved, 81 treated with anti PD-1 therapy. Median age was 14 years (range 2-18 years), 37 pts were ≤12 yrs. Overall, 38 CA patients received anti PD-1 in adjuvant setting, and the 3-year PFS and OS were 70.6 % and 81.1 %, respectively. Two patients received anti-PD-1 based neoadjuvant treatment, both had a pathologic complete response and remain disease free. Fifty-six received a systemic therapy for advanced disease and among them, 43 received anti PD-1-based therapy for advanced disease in 1st line, while 12 and 5 pts received a 2nd and 3rd line, respectively. Among patients receiving a 1st line therapy with anti PD-1 monotherapy the ORR was 25 %, and the 3-year OS was 34 %. Toxicities were consistent with previous studies in adult melanoma patients. Our study provides the first evidence of efficacy of anti PD-1 in CA melanoma patients and supports the use of anti PD-1 therapy in pts ≤18 years, included those <12 years.

Sections du résumé

BACKGROUND BACKGROUND
Data on the efficacy and safety of anti PD-1 antibodies in children and adolescents (CA) with melanoma are lacking. The aim of this study was to determine outcomes of CA melanoma patients receiving anti PD-1 antibodies.
METHODS METHODS
Melanoma patients ≤18 years treated with anti PD-1 were retrospectively retrieved from 15 academic centers. Information on histopathological diagnosis, surgical treatment, systemic therapy, objective response rate (ORR), safety profile was collected. Progression-free survival (PFS) and overall survival (OS) were assessed by Kaplan-Meier method.
RESULTS RESULTS
Between April 2016 and March 2024, 99 patients treated with systemic therapy were retrieved, 81 treated with anti PD-1 therapy. Median age was 14 years (range 2-18 years), 37 pts were ≤12 yrs. Overall, 38 CA patients received anti PD-1 in adjuvant setting, and the 3-year PFS and OS were 70.6 % and 81.1 %, respectively. Two patients received anti-PD-1 based neoadjuvant treatment, both had a pathologic complete response and remain disease free. Fifty-six received a systemic therapy for advanced disease and among them, 43 received anti PD-1-based therapy for advanced disease in 1st line, while 12 and 5 pts received a 2nd and 3rd line, respectively. Among patients receiving a 1st line therapy with anti PD-1 monotherapy the ORR was 25 %, and the 3-year OS was 34 %. Toxicities were consistent with previous studies in adult melanoma patients.
CONCLUSIONS CONCLUSIONS
Our study provides the first evidence of efficacy of anti PD-1 in CA melanoma patients and supports the use of anti PD-1 therapy in pts ≤18 years, included those <12 years.

Identifiants

pubmed: 39236557
pii: S0959-8049(24)00961-4
doi: 10.1016/j.ejca.2024.114305
pii:
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

114305

Informations de copyright

Copyright © 2024 The Authors. Published by Elsevier Ltd.. All rights reserved.

Déclaration de conflit d'intérêts

Declaration of Competing Interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper.

Auteurs

Mario Mandalà (M)

Unit of Medical Oncology, University of Perugia, Perugia, Italy. Electronic address: mario.mandala@unipg.it.

Andrea Ferrari (A)

Pediatric Oncology Unit, Fondazione IRCCS Istituto Nazionale Tumori, Milan, Italy; Department of Oncology and Hemato-oncology, University of Milan, Milan, Italy.

Ines B Brecht (IB)

Department of Pediatric Hematology and Oncology, University of Tubingen, Tubingen, Germany.

Karijn Pm Suijkerbuijk (KP)

Department of Medical Oncology, University Medical Center Utrecht, Utrecht University, Utrecht, Netherlands.

Linda Maschke (L)

Department of Pediatric Hematology and Oncology, University of Tubingen, Tubingen, Germany.

Diana Giannarelli (D)

Fondazione Policlinico Universitario A. Gemelli, IRCCS, Rome, Italy.

Alice Indini (A)

Melanoma Medical Oncology Unit, Department of Medical Oncology and Hematology, Fondazione IRCCS Istituto Nazionale Tumori, Milan, Italy.

Martina Ubaldi (M)

Unit of Medical Oncology, University of Perugia, Perugia, Italy.

Giulia Pecci (G)

Unit of Medical Oncology, University of Perugia, Perugia, Italy.

Victoria Atkinson (V)

Princess Alexandra Hospital, Brisbane, QLD, Australia.

Hildur Helgadottir (H)

Department of Oncology and Pathology, Karolinska Institutet, Skin Cancer Centrum, Theme Cancer, Karolinska University Hospital, Stockholm, Sweden.

Stefano Chiaravalli (S)

Pediatric Oncology Unit, Fondazione IRCCS Istituto Nazionale Tumori, Milan, Italy.

Naima Benannoune (N)

Gustave Roussy Cancer Campus, Villejuif, France.

Caroline Robert (C)

Gustave Roussy Cancer Campus, Villejuif, France.

Pawel Teterycz (P)

Maria Sklodowska-Curie National Research Institute of Oncology, Warsaw, Poland.

Piotr Rutkowski (P)

Maria Sklodowska-Curie National Research Institute of Oncology, Warsaw, Poland.

Susana Puig (S)

Dermatology Department, Hospital Clínic de Barcelona, Universitat de Barcelona, Institut de investigacions biomediques August Pi I Sunyer (IDIBAPS), Barcelona, Spain; Centro de Investigación BIomédica en Red de Enfermedades Raras (CIBERER), Instituto de Salud Carlos III, Barcelona, Spain.

Gabriele Madonna (G)

Melanoma, Cancer Immunotherapy and Development Therapeutics Unit, Istituto Nazionale Tumori IRCCS Fondazione G. Pascale, Naples, Italy.

Rejin Kebudi (R)

Istanbul University, Oncology Institute Pediatric Hematology-Oncology, Istanbul, Turkey.

Shirly Grynberg (S)

Ella Lemelbaum Institute for Immuno-Oncology and melanoma, Sheba Medical Center, Israel.

Lidia Mrb Arantes (LM)

Molecular Oncology Research Center, Barretos Cancer Hospital, Brazil.

Ewa Bien (E)

Department of Pediatrics Hematology and Oncology, Medical University of Gdański, Gdańsk, Poland.

Malgorzata Krawczyk (M)

Department of Pediatrics Hematology and Oncology, Medical University of Gdański, Gdańsk, Poland.

Maria Debora De Pasquale (MD)

Department of Hematology, Oncology and Stem Cell Transplantation, Bambino Gesù Children's Hospital IRCCS, Rome, Italy.

Miranda P Dierselhuis (MP)

Princess Máxima Center, Utrecht, Netherlands.

Daniela Massi (D)

Section of Anatomic Pathology, Department of Health Sciences, University of Florence, Florence, Italy; Department of Oral and Maxillofacial Surgery, New York University - College of Dentistry, New York, USA.

Georgina V Long (GV)

Melanoma Institute Australia, The University of Sydney, Sydney, NSW, Australia; Royal North Shore and Mater Hospitals, Australia.

Paolo Antonio Ascierto (PA)

Melanoma, Cancer Immunotherapy and Development Therapeutics Unit, Istituto Nazionale Tumori IRCCS Fondazione G. Pascale, Naples, Italy.

Alexander M M Eggermont (AMM)

Princess Máxima Center, Utrecht, Netherlands; University Medical Center Utrecht, Utrecht, Netherlands; Princess Máxima Center, Utrecht, Netherlands; Comprehensive Cancer Center Munich of the Technical University Munich and the Ludwig Maximilians University, Munich, Germany.

Classifications MeSH