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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
Drug delivery systems: application of liposomal anti-tumor agents to neuroectodermal cancer treatment.
Di Paolo D, Pastorino F, Brignole C, Marimpietri D, Loi M, Ponzoni M, Pagnan G
Tumori 2008 Mar-Apr;94(2):246-53. doi: 10.1177/030089160809400217. PMID: 18564613
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
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
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
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
Checkpoint inhibition in pediatric hematologic malignancies.
Davis KL, Agarwal AM, Verma AR
Pediatr Hematol Oncol 2017 Sep-Oct;34(6-7):379-394. Epub 2017 Nov 30 doi: 10.1080/08880018.2017.1383542. PMID: 29190182

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
Iodine-131 metaiodobenzylguanidine therapy for neuroblastoma: reports so far and future perspective.
Kayano D, Kinuya S
ScientificWorldJournal 2015;2015:189135. Epub 2015 Mar 22 doi: 10.1155/2015/189135. PMID: 25874239Free PMC Article
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

Therapy

A biobank of pediatric patient-derived-xenograft models in cancer precision medicine trial MAPPYACTS for relapsed and refractory tumors.
Marques Da Costa ME, Zaidi S, Scoazec JY, Droit R, Lim WC, Marchais A, Salmon J, Cherkaoui S, Morscher RJ, Laurent A, Malinge S, Mercher T, Tabone-Eglinger S, Goddard I, Pflumio F, Calvo J, Redini F, Entz-Werlé N, Soriano A, Villanueva A, Cairo S, Chastagner P, Moro M, Owens C, Casanova M, Hladun-Alvaro R, Berlanga P, Daudigeos-Dubus E, Dessen P, Zitvogel L, Lacroix L, Pierron G, Delattre O, Schleiermacher G, Surdez D, Geoerger B
Commun Biol 2023 Sep 18;6(1):949. doi: 10.1038/s42003-023-05320-0. PMID: 37723198Free PMC Article
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
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
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
Checkpoint inhibition in pediatric hematologic malignancies.
Davis KL, Agarwal AM, Verma AR
Pediatr Hematol Oncol 2017 Sep-Oct;34(6-7):379-394. Epub 2017 Nov 30 doi: 10.1080/08880018.2017.1383542. PMID: 29190182

Prognosis

A phase II trial of nifurtimox combined with topotecan and cyclophosphamide for refractory or relapsed neuroblastoma and medulloblastoma.
Eslin D, Zage PE, Bergendahl G, Lewis E, Roberts W, Kraveka J, Mitchell D, Isakoff MS, Rawwas J, Wada RK, Fluchel M, Brown VI, Ginn K, Higgins T, BeeravallyNagulapally A, Dykema K, Hanna G, Ferguson W, Saulnier Sholler GL
Int J Cancer 2023 Sep 1;153(5):1026-1034. Epub 2023 May 29 doi: 10.1002/ijc.34569. PMID: 37246577
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

Clinical prediction guides

Circulating tumor DNA sequencing of pediatric solid and brain tumor patients: An institutional feasibility study.
Mangum R, Reuther J, Sen Baksi K, Gandhi I, Zabriskie RC, Recinos A, Raesz-Martinez R, Lin FY, Potter SL, Sher AC, Kralik SF, Mohila CA, Chintagumpala MM, Muzny D, Hu J, Gibbs RA, Fisher KE, Bernini JC, Gill J, Griffin TC, Tomlinson GE, Vallance KL, Plon SE, Roy A, Parsons DW
Pediatr Hematol Oncol 2023;40(8):719-738. Epub 2023 Jun 27 doi: 10.1080/08880018.2023.2228837. PMID: 37366551Free PMC Article
A phase II trial of nifurtimox combined with topotecan and cyclophosphamide for refractory or relapsed neuroblastoma and medulloblastoma.
Eslin D, Zage PE, Bergendahl G, Lewis E, Roberts W, Kraveka J, Mitchell D, Isakoff MS, Rawwas J, Wada RK, Fluchel M, Brown VI, Ginn K, Higgins T, BeeravallyNagulapally A, Dykema K, Hanna G, Ferguson W, Saulnier Sholler GL
Int J Cancer 2023 Sep 1;153(5):1026-1034. Epub 2023 May 29 doi: 10.1002/ijc.34569. PMID: 37246577
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
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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