Roadmap for the next generation of Children's Oncology Group rhabdomyosarcoma trials

被引：1

作者：

Metts, Jonathan L. ^{[1
,2
]}

Aye, Jamie M. ^{[3
]}

Crane, Jacquelyn N. ^{[4
,5
]}

Oberoi, Sapna ^{[6
,7
]}

Balis, Frank M. ^{[4
]}

Bhatia, Smita ^{[8
]}

Bona, Kira ^{[9
,10
]}

Carleton, Bruce ^{[11
]}

Dasgupta, Roshni ^{[12
]}

Dela Cruz, Filemon S. ^{[13
]}

Greenzang, Katie A. ^{[9
,10
]}

Kaufman, Jonathan L. ^{[14
,15
]}

Linardic, Corinne M. ^{[16
,17
]}

Parsons, Susan K. ^{[18
,19
]}

Robertson-Tessi, Mark ^{[20
]}

Rudzinski, Erin R. ^{[21
,22
]}

Soragni, Alice ^{[23
,24
]}

Stewart, Elizabeth ^{[25
]}

Weigel, Brenda J. ^{[26
]}

Wolden, Suzanne L. ^{[27
]}

Weiss, Aaron R. ^{[28
]}

Venkatramani, Rajkumar ^{[29
]}

Heske, Christine M. ^{[30
]}

机构：

[1] Moffitt Comprehens Canc Ctr, Sarcoma Dept, Tampa, FL USA

[2] Johns Hopkins All Childrens Hosp, Canc & Blood Disorders Inst, St Petersburg, FL USA

[3] Univ Alabama Birmingham, Dept Pediat, Div Pediat Hematol & Oncol, Birmingham, AL USA

[4] Childrens Hosp Philadelphia, Div Oncol, Philadelphia, PA USA

[5] Univ Penn, Abramson Canc Ctr, Perelman Sch Med, Dept Pediat, Philadelphia, PA USA

[6] Canc Care Manitoba, Dept Pediat Hematol & Oncol, Winnipeg, MB, Canada

[7] Univ Manitoba, Dept Pediat & Child Hlth, Winnipeg, MB, Canada

[8] Univ Alabama Birmingham, Inst Canc Outcomes & Survivorship, Heersink Sch Med, Birmingham, AL USA

[9] Dana Farber Canc Inst, Dept Pediat Oncol, Boston, MA USA

[10] Dana Farber Canc Inst, Div Populat Sci, Boston, MA USA

[11] Univ British Columbia, Fac Med, Dept Pediat, Div Translat Therapeut, Vancouver, BC, Canada

[12] Univ Cincinnati, Cincinnati Childrens Hosp Med Ctr, Div Pediat Gen & Thorac Surg, Cincinnati, OH 45221 USA

[13] Mem Sloan Kettering Canc Ctr, Dept Pediat, New York, NY USA

[14] Emory Univ, Dept Hematol & Med Oncol, Atlanta, GA USA

[15] Patient Advocacy Comm, Childrens Oncol Grp, Monrovia, CA USA

[16] Duke Univ, Sch Med, Dept Pediat, Durham, NC USA

[17] Duke Univ, Sch Med, Dept Pharmacol & Canc Biol, Durham, NC USA

[18] Tufts Med Ctr, Inst Clin Res & Hlth Policy Studies, Boston, MA USA

[19] Tufts Med Ctr, Div Hematol Oncol, Boston, MA USA

[20] H Lee Moffitt Canc Ctr & Res Inst, Integrated Math Oncol Dept, Tampa, FL USA

[21] Seattle Childrens Hosp, Dept Lab Med & Pathol, Seattle, WA USA

[22] Univ Washington, Med Ctr, Seattle, WA USA

[23] Univ Calif Los Angeles, Dept Orthopaed Surg, Los Angeles, CA USA

[24] Univ Calif Los Angeles, Jonsson Comprehens Canc Ctr, Los Angeles, CA USA

[25] St Jude Childrens Res Hosp, Dept Oncol, Memphis, TN USA

[26] Univ Minnesota, Div Pediat Hematol & Oncol, Minneapolis, MN USA

[27] Mem Sloan Kettering Canc Ctr, Dept Radiat Oncol, New York, NY USA

[28] Maine Med Ctr, Dept Pediat, Portland, ME USA

[29] Baylor Coll Med, Texas Childrens Canc Ctr, Houston, TX USA

[30] NCI, Pediat Oncol Branch, Ctr Canc Res, Bethesda, MD USA

来源：

CANCER | 2024年 / 130卷 / 22期

关键词：

clinical trials; novel agents; pediatric; quality of life; rhabdomyosarcoma; PATIENT-REPORTED OUTCOMES; TISSUE SARCOMA COMMITTEE; RISK RHABDOMYOSARCOMA; PEDIATRIC-PATIENTS; 1ST RELAPSE; RADIOTHERAPY; VINCRISTINE; THERAPY; CLASSIFICATION; IMPLEMENTATION;

D O I：

10.1002/cncr.35457

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

Clinical trials conducted by the Intergroup Rhabdomyosarcoma (RMS) Study Group and the Children's Oncology Group have been pivotal to establishing current standards for diagnosis and therapy for RMS. Recent advancements in understanding the biology and clinical behavior of RMS have led to more nuanced approaches to diagnosis, risk stratification, and treatment. The complexities introduced by these advancements, coupled with the rarity of RMS, pose challenges to conducting large-scale phase 3 clinical trials to evaluate new treatment strategies for RMS. Given these challenges, systematic planning of future clinical trials in RMS is paramount to address pertinent questions regarding the therapeutic efficacy of drugs, biomarkers of response, treatment-related toxicity, and patient quality of life. Herein, the authors outline the proposed strategic approach of the Children's Oncology Group Soft Tissue Sarcoma Committee to the next generation of RMS clinical trials, focusing on five themes: improved novel agent identification and preclinical to clinical translation, more efficient trial development and implementation, expanded opportunities for knowledge generation during trials, therapeutic toxicity reduction and quality of life, and patient engagement.

引用

页码：3785 / 3796

页数：12

共 93 条

[41] Children's Oncology Group's 2023 blueprint for research: Radiation oncology [J].

Kalapurakal, John A. L. ;

Wolden, Suzanne L. ;

Haas-Kogan, Daphne N. ;

Laack, Nadia N. ;

Hua, Chia-ho C. ;

Paulino, Arnold C. E. ;

Hill-Kayser, Christine E. S. ;

Hoppe, Bradford S. J. ;

Fitzgerald, Thomas J. ;

COG Radiation Oncology Discipline Com .

PEDIATRIC BLOOD & CANCER, 2023, 70

[42] National Cancer Institute Pediatric Preclinical Testing Program: Model Description for In Vitro Cytotoxicity Testing [J].

Kang, Min H. ;

Smith, Malcolm A. ;

Morton, Christopher L. ;

Keshelava, Nino ;

Houghton, Peter J. ;

Reynolds, C. Patrick .

PEDIATRIC BLOOD & CANCER, 2011, 56 (02) :239-249

[43] Patient advocates' role in clinical trials [J].

Katz, Mira L. ;

Archer, Laura E. ;

Peppercorn, Jeffrey M. ;

Kereakoglow, Sandra ;

Collyar, Deborah E. ;

Burstein, Harold J. ;

Schilsky, Richard L. ;

Partridge, Ann H. .

CANCER, 2012, 118 (19) :4801-4805

[44] Minimal adjuvant chemotherapy for children with hepatoblastoma resected at diagnosis (AHEP0731): a Children's Oncology Group, multicentre, phase 3 trial [J].

Katzenstein, Howard M. d ;

Langham, Max R. ;

Malogolowkin, Marcio H. ;

Krailo, Mark D. ;

Towbin, Alexander J. ;

McCarville, Mary Beth ;

Finegold, Milton J. ;

Ranganathan, Sarangarajan ;

Dunn, Stephen ;

McGahren, Eugene D. ;

Tiao, Gregory M. ;

O'Neill, Allison F. ;

Qayed, Muna ;

Furman, Wayne L. ;

Xia, Caihong ;

Rodriguez-Galindo, Carlos ;

Meyers, Rebecka L. .

LANCET ONCOLOGY, 2019, 20 (05) :719-727

[45] Focusing on Core Patient-Reported Outcomes in Cancer Clinical Trials: Symptomatic Adverse Events, Physical Function, and Disease-Related Symptoms [J].

Kluetz, Paul G. ;

Slagle, Ashley ;

Papadopoulos, Elektra J. ;

Johnson, Laura Lee ;

Donoghue, Martha ;

Kwitkowski, Virginia E. ;

Chen, Wen-Hung ;

Sridhara, Rajeshwari ;

Farrell, Ann T. ;

Keegan, Patricia ;

Kim, Geoffrey ;

Pazdur, Richard .

CLINICAL CANCER RESEARCH, 2016, 22 (07) :1553-1558

[46]

Kurzrock Razelle, 2021, Am Soc Clin Oncol Educ Book, V41, pe133, DOI [10.1200/edbk_319783, 10.1200/EDBK_319783]

[47] Cell-Free DNA as a Diagnostic and Prognostic Biomarker in Pediatric Rhabdomyosarcoma [J].

Lak, Nathalie S. M. ;

van Zogchel, Lieke M. J. ;

Zappeij-Kannegieter, Lily ;

Javadi, Ahmad ;

van Paemel, Ruben ;

Vandeputte, Charlotte ;

De Preter, Katleen ;

De Wilde, Bram ;

Chicard, Mathieu ;

Iddir, Yasmine ;

Schleiermacher, Gudrun ;

Ruhen, Olivia ;

Shipley, Janet ;

Fiocco, Marta ;

Merks, Johannes H. M. ;

van Noesel, Max M. ;

van der Schoot, C. Ellen ;

Tytgat, Godelieve A. M. ;

Stutterheim, Janine .

JCO PRECISION ONCOLOGY, 2023, 7

[48] Improving Risk Stratification for Pediatric Patients with Rhabdomyosarcoma by Molecular Detection of Disseminated Disease [J].

Lak, Nathalie S. M. ;

Voormanns, Timon L. ;

Zappeij-Kannegieter, Lily ;

van Zogchel, Lieke M. J. ;

Fiocco, Marta ;

van Noesel, Max M. ;

Merks, Johannes H. M. ;

Van der Schoot, C. Ellen ;

Tytgat, Godelieve A. M. ;

Stutterheim, Janine .

CLINICAL CANCER RESEARCH, 2021, 27 (20) :5576-5585

[49] Relationship between tumor response at therapy completion and prognosis in patients with Group III rhabdomyosarcoma: A report from the Children's Oncology Group [J].

Lautz, Timothy B. ;

Chi, Yueh-Yun ;

Tian, Jing ;

Gupta, Abha A. ;

Wolden, Suzanne L. ;

Routh, Jonathan C. ;

Casey, Dana L. ;

Dasgupta, Roshni ;

Hawkins, Douglas S. ;

Rodeberg, David A. .

INTERNATIONAL JOURNAL OF CANCER, 2020, 147 (05) :1419-1426

[50] Symptom Monitoring in Pediatric Oncology Using Patient-Reported Outcomes: Why, How, and Where Next [J].

Leahy, Allison Barz ;

Feudtner, Chris ;

Basch, Ethan .

PATIENT-PATIENT CENTERED OUTCOMES RESEARCH, 2018, 11 (02) :147-153

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