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

被引：7

作者：

Mandala, Mario ^{[1
]}

Ferrari, Andrea ^{[2
,3
]}

Brecht, Ines B. ^{[4
]}

Suijkerbuijk, Karijn P. M. ^{[5
]}

Maschke, Linda ^{[4
,6
]}

Giannarelli, Diana ^{[5
]}

Indini, Alice ^{[7
]}

Ubaldi, Martina ^{[1
]}

Pecci, Giulia ^{[1
]}

Atkinson, Victoria ^{[8
]}

Helgadottir, Hildur ^{[9
]}

Chiaravalli, Stefano ^{[2
]}

Benannoune, Naima ^{[10
]}

Robert, Caroline ^{[10
]}

Teterycz, Pawel ^{[11
]}

Rutkowski, Piotr ^{[11
]}

Puig, Susana ^{[12
,13
]}

Madonna, Gabriele ^{[14
]}

Kebudi, Rejin ^{[15
]}

Grynberg, Shirly ^{[16
]}

Arantes, Lidia M. R. B. ^{[17
]}

Bien, Ewa ^{[18
]}

Krawczyk, Malgorzata ^{[18
]}

Pasquale, Maria Debora De ^{[19
]}

Dierselhuis, Miranda P. ^{[20
]}

Massi, Daniela ^{[21
,22
]}

V. Long, Georgina ^{[23
]}

Ascierto, Paolo Antonio ^{[13
]}

Eggermont, Alexander M. M. ^{[20
,24
,25
,26
]}

机构：

[1] Univ Perugia, Unit Med Oncol, Perugia, Italy

[2] Fdn IRCCS Ist Nazl Tumori, Pediat Oncol Unit, Milan, Italy

[3] Univ Milan, Dept Oncol & Hematooncol, Milan, Italy

[4] Univ Tubingen, Dept Pediat Hematol & Oncol, Tubingen, Germany

[5] Univ Utrecht, Univ Med Ctr Utrecht, Dept Med Oncol, Utrecht, Netherlands

[6] IRCCS, Fdn Policlin Univ A Gemelli, Rome, Italy

[7] Fdn IRCCS Ist Nazl Tumori, Dept Med Oncol & Hematol, Melanoma Med Oncol Unit, Milan, Italy

[8] Princess Alexandra Hosp, Brisbane, Qld, Australia

[9] Karolinska Univ Hosp, Karolinska Inst, Dept Oncol & Pathol, Skin Canc Ctr,Theme Canc, Stockholm, Sweden

[10] Gustave Roussy Canc Campus, Villejuif, France

[11] Maria Sklodowska Curie Natl Res Inst Oncol, Warsaw, Poland

[12] Univ Barcelona, Hosp Clin Barcelona, Inst Invest Biomed August Pi & Sunyer IDIBAPS, Dermatol Dept, Barcelona, Spain

[13] Inst Salud Carlos III, Ctr Invest BIomed Red Enfermedades Raras CIBERER, Barcelona, Spain

[14] Ist Nazl Tumori IRCCS Fdn G Pascale, Melanoma Canc Immunotherapy & Dev Therapeut Unit, Naples, Italy

[15] Istanbul Univ, Oncol Inst Pediat Hematol Oncol, Istanbul, Turkiye

[16] Sheba Med Ctr, Ella Lemelbaum Inst Immunooncol & Melanoma, Ramat Gan, Israel

[17] Barretos Canc Hosp, Mol Oncol Res Ctr, Sao Paulo, Brazil

[18] Med Univ Gdanski, Dept Pediat Hematol & Oncol, Gdansk, Poland

[19] Bambino Gesu Childrens Hosp IRCCS, Dept Hematol Oncol & Stem Cell Transplantat, Rome, Italy

[20] Princess Maxima Ctr, Utrecht, Netherlands

[21] Univ Florence, Dept Hlth Sci, Sect Anat Pathol, Florence, Italy

[22] NYU, Coll Dent, Dept Oral & Maxillofacial Surg, New York, NY USA

[23] Univ Sydney, Melanoma Inst Australia, Sydney, NSW, Australia

[24] Univ Med Ctr Utrecht, Utrecht, Netherlands

[25] Ludwig Maximilians Univ Munchen, Comprehens Canc Ctr Munich Tech Univ Munich, Munich, Germany

[26] Royal North Shore & Mater Hosp, Sydney, Australia

来源：

EUROPEAN JOURNAL OF CANCER | 2024年 / 211卷

关键词：

Melanoma; Child; Adolescent; Anti PD-1; Outcome; Safety; RESECTED STAGE-III; SINGLE-ARM; OPEN-LABEL; NIVOLUMAB; PEMBROLIZUMAB; IPILIMUMAB;

D O I：

10.1016/j.ejca.2024.114305

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

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: 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: 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: 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.

引用

页数：6

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