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Refractory Childhood Malignant Solid Neoplasm

MedGen UID:
1644994
•Concept ID:
C4688334
•
Neoplastic Process
Synonyms: Childhood Refractory Malignant Solid Neoplasm; Childhood Refractory Malignant Solid Tumor

Definition

A childhood malignant solid neoplasm that does not respond to treatment. [from NCI]

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  
  • CROGVRefractory Childhood Malignant Solid Neoplasm

Professional guidelines

PubMed

Personalized Treatment Response Assessment for Rare Childhood Tumors Using Microcalorimetry-Exemplified by Use of Carbonic Anhydrase IX and Aquaporin 1 Inhibitors.
Gros SJ, Holland-Cunz SG, Supuran CT, Braissant O
Int J Mol Sci 2019 Oct 9;20(20) doi: 10.3390/ijms20204984. PMID: 31600976Free PMC Article
Second Neoplasms in Children Following a Treatment for Acute Leukemia and/or Lymphoma: 29 Years of Experience in a Single Institution in Argentina.
Felice MS, Rossi JG, Alonso CN, Rubio P, Gallego MS, Galluzzo ML, Lubieniecki F, Gutiérrez G, Guitter MR, Alderete DH, Rose AB, Cacciavillano WD, Herzovich V, Alfaro EM, Sánchez La Rosa CG, Millán N, Chantada GL, Figueroa Turienzo CM, Zubizarreta PA
J Pediatr Hematol Oncol 2017 Nov;39(8):e406-e412. doi: 10.1097/MPH.0000000000000971. PMID: 28945661
Epipodophyllotoxins in the treatment of childhood cancer.
Rivera GK, Pui CH, Santana VM, Pratt CB, Crist WM
Cancer Chemother Pharmacol 1994;34 Suppl:S89-95. doi: 10.1007/BF00684870. PMID: 8070034

Recent clinical studies

Etiology

A Phase I/II Trial of Nivolumab plus Ipilimumab in Children and Young Adults with Relapsed/Refractory Solid Tumors: A Children's Oncology Group Study ADVL1412.
Davis KL, Fox E, Isikwei E, Reid JM, Liu X, Minard CG, Voss S, Berg SL, Weigel BJ, Mackall CL
Clin Cancer Res 2022 Dec 1;28(23):5088-5097. doi: 10.1158/1078-0432.CCR-22-2164. PMID: 36190525Free PMC Article
Actionable Tumor Alterations and Treatment Protocol Enrollment of Pediatric and Young Adult Patients With Refractory Cancers in the National Cancer Institute-Children's Oncology Group Pediatric MATCH Trial.
Parsons DW, Janeway KA, Patton DR, Winter CL, Coffey B, Williams PM, Roy-Chowdhuri S, Tsongalis GJ, Routbort M, Ramirez NC, Saguilig L, Piao J, Alonzo TA, Berg SL, Fox E, Hawkins DS, Abrams JS, Mooney M, Takebe N, Tricoli JV, Seibel NL; NCI-COG Pediatric MATCH Team
J Clin Oncol 2022 Jul 10;40(20):2224-2234. Epub 2022 Mar 30 doi: 10.1200/JCO.21.02838. PMID: 35353553Free PMC Article
Nivolumab in children and young adults with relapsed or refractory solid tumours or lymphoma (ADVL1412): a multicentre, open-label, single-arm, phase 1-2 trial.
Davis KL, Fox E, Merchant MS, Reid JM, Kudgus RA, Liu X, Minard CG, Voss S, Berg SL, Weigel BJ, Mackall CL
Lancet Oncol 2020 Apr;21(4):541-550. Epub 2020 Mar 17 doi: 10.1016/S1470-2045(20)30023-1. PMID: 32192573Free PMC Article
The continuous rediscovery and the benefit-risk ratio of thioguanine, a comprehensive review.
Bayoumy AB, Simsek M, Seinen ML, Mulder CJJ, Ansari A, Peters GJ, De Boer NK
Expert Opin Drug Metab Toxicol 2020 Feb;16(2):111-123. Epub 2020 Feb 24 doi: 10.1080/17425255.2020.1719996. PMID: 32090622
Too many targets, not enough patients: rethinking neuroblastoma clinical trials.
Fletcher JI, Ziegler DS, Trahair TN, Marshall GM, Haber M, Norris MD
Nat Rev Cancer 2018 Jun;18(6):389-400. doi: 10.1038/s41568-018-0003-x. PMID: 29632319

Diagnosis

Genomic and Transcriptomic Analysis of Relapsed and Refractory Childhood Solid Tumors Reveals a Diverse Molecular Landscape and Mechanisms of Immune Evasion.
Byron SA, Hendricks WPD, Nagulapally AB, Kraveka JM, Ferguson WS, Brown VI, Eslin DE, Mitchell D, Cornelius A, Roberts W, Isakoff MS, Oesterheld JE, Wada RK, Rawwas J, Neville K, Zage PE, Harrod VL, Bergendahl G, VanSickle E, Dykema K, Bond J, Chou HC, Wei JS, Wen X, Reardon HV, Roos A, Nasser S, Izatt T, Enriquez D, Hegde AM, Cisneros F, Christofferson A, Turner B, Szelinger S, Keats JJ, Halperin RF, Khan J, Saulnier Sholler GL, Trent JM
Cancer Res 2021 Dec 1;81(23):5818-5832. Epub 2021 Oct 5 doi: 10.1158/0008-5472.CAN-21-1033. PMID: 34610968Free PMC Article
Relapsing intracranial germ cell tumours warrant retreatment.
Callec L, Lardy-Cleaud A, Guerrini-Rousseau L, Alapetite C, Vignon L, Chastagner P, Frappaz D, Faure-Conter C
Eur J Cancer 2020 Sep;136:186-194. Epub 2020 Jul 22 doi: 10.1016/j.ejca.2020.06.012. PMID: 32711377
Too many targets, not enough patients: rethinking neuroblastoma clinical trials.
Fletcher JI, Ziegler DS, Trahair TN, Marshall GM, Haber M, Norris MD
Nat Rev Cancer 2018 Jun;18(6):389-400. doi: 10.1038/s41568-018-0003-x. PMID: 29632319
Neuroblastoma and other neuroendocrine tumors.
Howman-Giles R, Shaw PJ, Uren RF, Chung DK
Semin Nucl Med 2007 Jul;37(4):286-302. doi: 10.1053/j.semnuclmed.2007.02.009. PMID: 17544628
Epipodophyllotoxins in the treatment of childhood cancer.
Rivera GK, Pui CH, Santana VM, Pratt CB, Crist WM
Cancer Chemother Pharmacol 1994;34 Suppl:S89-95. doi: 10.1007/BF00684870. PMID: 8070034

Therapy

A Phase I/II Trial of Nivolumab plus Ipilimumab in Children and Young Adults with Relapsed/Refractory Solid Tumors: A Children's Oncology Group Study ADVL1412.
Davis KL, Fox E, Isikwei E, Reid JM, Liu X, Minard CG, Voss S, Berg SL, Weigel BJ, Mackall CL
Clin Cancer Res 2022 Dec 1;28(23):5088-5097. doi: 10.1158/1078-0432.CCR-22-2164. PMID: 36190525Free PMC Article
Actionable Tumor Alterations and Treatment Protocol Enrollment of Pediatric and Young Adult Patients With Refractory Cancers in the National Cancer Institute-Children's Oncology Group Pediatric MATCH Trial.
Parsons DW, Janeway KA, Patton DR, Winter CL, Coffey B, Williams PM, Roy-Chowdhuri S, Tsongalis GJ, Routbort M, Ramirez NC, Saguilig L, Piao J, Alonzo TA, Berg SL, Fox E, Hawkins DS, Abrams JS, Mooney M, Takebe N, Tricoli JV, Seibel NL; NCI-COG Pediatric MATCH Team
J Clin Oncol 2022 Jul 10;40(20):2224-2234. Epub 2022 Mar 30 doi: 10.1200/JCO.21.02838. PMID: 35353553Free PMC Article
Nivolumab in children and young adults with relapsed or refractory solid tumours or lymphoma (ADVL1412): a multicentre, open-label, single-arm, phase 1-2 trial.
Davis KL, Fox E, Merchant MS, Reid JM, Kudgus RA, Liu X, Minard CG, Voss S, Berg SL, Weigel BJ, Mackall CL
Lancet Oncol 2020 Apr;21(4):541-550. Epub 2020 Mar 17 doi: 10.1016/S1470-2045(20)30023-1. PMID: 32192573Free PMC Article
Efficacy and Safety of Dabrafenib in Pediatric Patients with BRAF V600 Mutation-Positive Relapsed or Refractory Low-Grade Glioma: Results from a Phase I/IIa Study.
Hargrave DR, Bouffet E, Tabori U, Broniscer A, Cohen KJ, Hansford JR, Geoerger B, Hingorani P, Dunkel IJ, Russo MW, Tseng L, Dasgupta K, Gasal E, Whitlock JA, Kieran MW
Clin Cancer Res 2019 Dec 15;25(24):7303-7311. doi: 10.1158/1078-0432.CCR-19-2177. PMID: 31811016
Too many targets, not enough patients: rethinking neuroblastoma clinical trials.
Fletcher JI, Ziegler DS, Trahair TN, Marshall GM, Haber M, Norris MD
Nat Rev Cancer 2018 Jun;18(6):389-400. doi: 10.1038/s41568-018-0003-x. PMID: 29632319

Prognosis

Macrophage-mediated anti-tumor immunity against high-risk neuroblastoma.
Tang XX, Shimada H, Ikegaki N
Genes Immun 2022 Jun;23(3-4):129-140. Epub 2022 May 7 doi: 10.1038/s41435-022-00172-w. PMID: 35525858Free PMC Article
Genomic and Transcriptomic Analysis of Relapsed and Refractory Childhood Solid Tumors Reveals a Diverse Molecular Landscape and Mechanisms of Immune Evasion.
Byron SA, Hendricks WPD, Nagulapally AB, Kraveka JM, Ferguson WS, Brown VI, Eslin DE, Mitchell D, Cornelius A, Roberts W, Isakoff MS, Oesterheld JE, Wada RK, Rawwas J, Neville K, Zage PE, Harrod VL, Bergendahl G, VanSickle E, Dykema K, Bond J, Chou HC, Wei JS, Wen X, Reardon HV, Roos A, Nasser S, Izatt T, Enriquez D, Hegde AM, Cisneros F, Christofferson A, Turner B, Szelinger S, Keats JJ, Halperin RF, Khan J, Saulnier Sholler GL, Trent JM
Cancer Res 2021 Dec 1;81(23):5818-5832. Epub 2021 Oct 5 doi: 10.1158/0008-5472.CAN-21-1033. PMID: 34610968Free PMC Article
CD146 is a potential immunotarget for neuroblastoma.
Obu S, Umeda K, Ueno H, Sonoda M, Tasaka K, Ogata H, Kouzuki K, Nodomi S, Saida S, Kato I, Hiramatsu H, Okamoto T, Ogawa E, Okajima H, Morita K, Kamikubo Y, Kawaguchi K, Watanabe K, Iwafuchi H, Yagyu S, Iehara T, Hosoi H, Nakahata T, Adachi S, Uemoto S, Heike T, Takita J
Cancer Sci 2021 Nov;112(11):4617-4626. Epub 2021 Sep 30 doi: 10.1111/cas.15124. PMID: 34464480Free PMC Article
Future directions in risk stratification and therapy for advanced pediatric genitourinary rhabdomyosarcoma.
Harel M, Ferrer FA, Shapiro LH, Makari JH
Urol Oncol 2016 Feb;34(2):103-15. Epub 2015 Oct 28 doi: 10.1016/j.urolonc.2015.09.013. PMID: 26519985
Neuroblastoma and other neuroendocrine tumors.
Howman-Giles R, Shaw PJ, Uren RF, Chung DK
Semin Nucl Med 2007 Jul;37(4):286-302. doi: 10.1053/j.semnuclmed.2007.02.009. PMID: 17544628

Clinical prediction guides

A Phase I/II Trial of Nivolumab plus Ipilimumab in Children and Young Adults with Relapsed/Refractory Solid Tumors: A Children's Oncology Group Study ADVL1412.
Davis KL, Fox E, Isikwei E, Reid JM, Liu X, Minard CG, Voss S, Berg SL, Weigel BJ, Mackall CL
Clin Cancer Res 2022 Dec 1;28(23):5088-5097. doi: 10.1158/1078-0432.CCR-22-2164. PMID: 36190525Free PMC Article
First phase 1 clinical study of olaparib in pediatric patients with refractory solid tumors.
Takagi M, Ogawa C, Iehara T, Aoki-Nogami Y, Ishibashi E, Imai M, Kimura T, Nagata M, Yasuhara M, Masutani M, Yoshimura K, Tomizawa D, Ogawa A, Yonemori K, Morishita A, Miyamoto S, Takita J, Kihara T, Nobori K, Hasebe K, Miya F, Ikeda S, Shioda Y, Matsumoto K, Fujimura J, Mizutani S, Morio T, Hosoi H, Koike R
Cancer 2022 Aug 1;128(15):2949-2957. Epub 2022 May 20 doi: 10.1002/cncr.34270. PMID: 35593736
Genomic and Transcriptomic Analysis of Relapsed and Refractory Childhood Solid Tumors Reveals a Diverse Molecular Landscape and Mechanisms of Immune Evasion.
Byron SA, Hendricks WPD, Nagulapally AB, Kraveka JM, Ferguson WS, Brown VI, Eslin DE, Mitchell D, Cornelius A, Roberts W, Isakoff MS, Oesterheld JE, Wada RK, Rawwas J, Neville K, Zage PE, Harrod VL, Bergendahl G, VanSickle E, Dykema K, Bond J, Chou HC, Wei JS, Wen X, Reardon HV, Roos A, Nasser S, Izatt T, Enriquez D, Hegde AM, Cisneros F, Christofferson A, Turner B, Szelinger S, Keats JJ, Halperin RF, Khan J, Saulnier Sholler GL, Trent JM
Cancer Res 2021 Dec 1;81(23):5818-5832. Epub 2021 Oct 5 doi: 10.1158/0008-5472.CAN-21-1033. PMID: 34610968Free PMC Article
Relapsing intracranial germ cell tumours warrant retreatment.
Callec L, Lardy-Cleaud A, Guerrini-Rousseau L, Alapetite C, Vignon L, Chastagner P, Frappaz D, Faure-Conter C
Eur J Cancer 2020 Sep;136:186-194. Epub 2020 Jul 22 doi: 10.1016/j.ejca.2020.06.012. PMID: 32711377
Nivolumab in children and young adults with relapsed or refractory solid tumours or lymphoma (ADVL1412): a multicentre, open-label, single-arm, phase 1-2 trial.
Davis KL, Fox E, Merchant MS, Reid JM, Kudgus RA, Liu X, Minard CG, Voss S, Berg SL, Weigel BJ, Mackall CL
Lancet Oncol 2020 Apr;21(4):541-550. Epub 2020 Mar 17 doi: 10.1016/S1470-2045(20)30023-1. PMID: 32192573Free PMC Article

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