[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"detail-sidebar-cat-0-en-105":3,"doc-seo-372790-105":59,"doc-detail-372790-en":131},{"code":4,"msg":5,"data":6},0,"success",[7,13,18,23,28,33,38,43,48,51,55],{"id":8,"doc_module":4,"doc_module_name":9,"category_name":10,"show_sort_weight":11,"slug":12},1,"Document","Story & Novel",90,"story-novel",{"id":14,"doc_module":4,"doc_module_name":9,"category_name":15,"show_sort_weight":16,"slug":17},2,"Literature",80,"literature",{"id":19,"doc_module":4,"doc_module_name":9,"category_name":20,"show_sort_weight":21,"slug":22},4,"Exam",70,"exam",{"id":24,"doc_module":4,"doc_module_name":9,"category_name":25,"show_sort_weight":26,"slug":27},5,"Comic",60,"comic",{"id":29,"doc_module":4,"doc_module_name":9,"category_name":30,"show_sort_weight":31,"slug":32},6,"Technology",50,"technology",{"id":34,"doc_module":4,"doc_module_name":9,"category_name":35,"show_sort_weight":36,"slug":37},7,"Healthcare",40,"healthcare",{"id":39,"doc_module":4,"doc_module_name":9,"category_name":40,"show_sort_weight":41,"slug":42},8,"Research & Report",30,"research-report",{"id":44,"doc_module":4,"doc_module_name":9,"category_name":45,"show_sort_weight":46,"slug":47},9,"Religion & Spirituality",20,"religion-spirituality",{"id":46,"doc_module":4,"doc_module_name":9,"category_name":49,"show_sort_weight":46,"slug":50},"World Cup","world-cup",{"id":52,"doc_module":4,"doc_module_name":9,"category_name":53,"show_sort_weight":52,"slug":54},10,"Lifestyle","lifestyle",{"id":56,"doc_module":4,"doc_module_name":9,"category_name":57,"show_sort_weight":24,"slug":58},19,"General","general",{"code":4,"msg":60,"data":61},"ok",{"site_id":62,"language":63,"slug":64,"title":65,"keywords":66,"description":67,"schema_data":68,"social_meta":124,"head_meta":126,"extra_data":128,"updated_unix":130},105,"en","rare-but-not-forgotten-therapeutic-advancements-for-rare-childhood-cancers","Rare but not forgotten - Therapeutic advancements for rare childhood cancers","","Cancers in children account for less than 1% of all new cancer cases, and only a subset qualifies as particularly rare in both children and adults. This review synthesizes multi-institutional pediatric trial progress and coordinated pediatric–adult research, emphasizing immunotherapies, molecularly targeted therapies, and radioligands. Coverage includes immune checkpoint inhibitor approvals across rare pediatric tumor types, NTRK inhibitors for NTRK-fusion solid tumors, ALK inhibition for ALK-positive inflammatory myofibroblastic tumors, and LUATHERA for somatostatin receptor-positive neuroendocrine tumors, alongside adult data informing pediatric rare-cancer management. Persistent challenges include small patient numbers, trial power limitations, and limited financial incentives, underscoring the need for continued collaboration to improve survival and reduce late effects.",{"@graph":69,"@context":123},[70,84,106],{"@type":71,"itemListElement":72},"BreadcrumbList",[73,77,79,82],{"item":74,"name":75,"@type":76,"position":8},"https://docshare.wps.com","Home","ListItem",{"item":78,"name":9,"@type":76,"position":14},"https://docshare.wps.com/document/",{"item":80,"name":35,"@type":76,"position":81},"https://docshare.wps.com/document/healthcare/",3,{"item":83,"name":65,"@type":76,"position":19},"https://docshare.wps.com/document/rare-but-not-forgotten-therapeutic-advancements-for-rare-childhood-cancers/372790/",{"url":83,"name":65,"@type":85,"image":86,"author":91,"headline":65,"publisher":94,"fileFormat":97,"inLanguage":63,"description":67,"dateModified":98,"datePublished":99,"encodingFormat":97,"isAccessibleForFree":100,"interactionStatistic":101},"DigitalDocument",{"url":87,"@type":88,"width":89,"height":90},"https://docshare.wps.com/thumbnails/rare-but-not-forgotten-therapeutic-advancements-for-rare-childhood-cancers/372790.png","ImageObject",300,407,{"name":92,"@type":93},"awa","Person",{"url":74,"name":95,"@type":96},"DocShare","Organization","application/pdf","2026-11-01","2026-09-23",true,{"@type":102,"interactionType":103,"userInteractionCount":105},"InteractionCounter",{"@type":104},"ViewAction",11,{"@type":107,"mainEntity":108},"FAQPage",[109,115,119],{"name":110,"@type":111,"acceptedAnswer":112},"Which types of treatment advancements does the review focus on for rare childhood cancers?","Question",{"text":113,"@type":114},"The review focuses on immunotherapies, molecularly targeted therapies, and radioligands, highlighting progress beyond conventional chemotherapy.","Answer",{"name":116,"@type":111,"acceptedAnswer":117},"What therapeutic areas and drug classes are discussed as examples of immunotherapy in rare pediatric cancers?",{"text":118,"@type":114},"Examples include immune checkpoint inhibitors and adoptive cell therapies, including blinatumomab, tisagenlecleucel, and dinutuximab, each transforming treatment in specific pediatric cancers.",{"name":120,"@type":111,"acceptedAnswer":121},"What major challenges limit progress in rare pediatric cancer clinical trials?",{"text":122,"@type":114},"The review identifies limited patient numbers for efficient, well-powered trials and a lack of financial incentives, requiring ongoing coordinated efforts to improve survival and minimize late effects.","https://schema.org",{"og:url":83,"og:type":125,"og:title":65,"og:site_name":95,"og:description":67},"article",{"robots":127,"canonical":83},"index,follow",{"doc_id":129,"site_id":62},372790,1790588240,{"code":4,"msg":5,"data":132},{"doc_id":129,"user_id":133,"nickname":92,"user_avatar":134,"doc_module":4,"category_id":34,"category_name":35,"doc_title":65,"doc_description":67,"doc_content":135,"file_id":136,"file_url":137,"file_type":138,"file_size":139,"view_count":105,"is_deleted":4,"is_public":8,"is_downloadable":8,"audit_status":8,"page_count":140,"language":141,"language_code":63,"site_id":62,"html_lang":63,"table_of_contents":142,"faqs":143,"seo_title":144,"seo_description":67,"update_tm":145,"read_time":146},3985747858093,"https://ap-avatar.wpscdn.com/davatar_3d24733baf745e90a7e4bdd5f77d97b2","Rare but not forgotten: Therapeutic  \nadvancements for rare childhood cancers  \nJacquelyn N. Crane, 1,2 Urania Dagalakis,3 Robyn D. Gartrell,4 Kris Ann P. Schultz,5 and Theodore W. Laetsch 1,2  \n1Department of Pediatrics, Abramson Cancer Center, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA; 2Division of Oncology, Center for Childhood Cancer Research, Children’s Hospital of Philadelphia, Philadelphia, PA 19104, USA; 3Division of Oncology, Cancer and Blood Disorders Center, Children’s National Hospital, Washington, DC 20010, USA; 4Department of Oncology, Division of Pediatric Oncology, Johns Hopkins University School of Medicine, Baltimore, MD 21218, USA; 5Cancer and Blood Disorders, Children’s Minnesota, Minneapolis, MN 55404, USA  \nCancers in children represent less than 1% of all new cases of cancer. While all cancers are rare during childhood, certain types are particularly rare. Multi-institutional pediatric trials and coordinated efforts between pediatric and adult groups have resulted in recent key advances in the treatment of certain rare pediatric cancers. This article reviews these advances with a focus on immunotherapies, molecularly targeted therapies, and radioligands. This includes work that led to the FDA approvals of immune checkpoint inhibitors in multiple rare pediatric tumor types, the NTRK inhibitors larotrectinib, entrectinib, andrepotrectinib for children and adults with solid tumors with NTRK fusions, the ALK inhibitor crizotinib in children and adults with ALK-positive inflammatory myofibroblastic tumors, and the radioligand LUATHERA for adolescents and adults with somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors. Additionally, key data from adult studies which have informed the management ofpediatric rare cancers are reviewed. Despite these advances, the study of rare pediatric cancers faces multiple challenges including a limited number ofpatients for efficient and well-powered clinical trials and a dearth of financial incentives. Ongoing, coordinated efforts are needed to continue the advancement of novel treatments and improve survival and minimize late effects.  \nINTRODUCTION  \nThere are approximately 1,800,000 new cases of cancer diagnosed each year in the United States and about 15,000 of these occur in children less than 20 years of age. 1 Thus, pediatric cancers represent less than 1% of all cancer cases. While all childhood cancers are rare, there are varying definitions of what constitutes a “rare” pediatric cancer.2–5 This article will focus on cancers that are particularly rare in both children and adults and those that are particularly rare in children despite being common in adults. This includes but is not limited to carcinomas (nasopharyngeal carcinoma [NPC], thyroid carcinoma, adrenocortical carcinoma [ACC], and colorectal carcinoma [CRC]), non-rhabdomyosarcoma soft tissue sarcomas (alveolar soft part sarcoma [ASPS], infantile fibrosarcoma, and synovial sarcoma), soft tissue tumors with intermediate biologic potential (inflammatory myofibroblastic tumors [IMT]), melanoma, neuroendocrine tumors, and pleuropulmonary blastoma (PPB) .  \nSurvival for patients with pediatric cancers has improved over the past several decades. However, this progress has largely been restricted to the more common pediatric cancer types with modest to no improvement in many other pediatric cancer types with some important exceptions.6,7 Additionally, the morbidity of childhood cancer and its treatments remains significant with the majority of childhood cancer survivors experiencing chronic health care conditions and late effects.8,9 There is great interest in novel treatment approaches to improve survival and limit long term treatment effects. Fortunately, there have been several recent key advancementsin the treatments of certain rare pediatric cancers. Herein, we review these advancements with a focus on treatment approaches other than conventio","cbCaimi9kkxUojrL","https://ap.wps.com/l/cbCaimi9kkxUojrL","pdf",2286489,13,"English","# Introduction\n# Immunotherapies\n## Immune checkpoint inhibitors overview and use in pediatric rare cancers\n# Molecularly targeted therapies\n# Radioligands\n# Challenges and future directions","[{\"question\":\"Which types of treatment advancements does the review focus on for rare childhood cancers?\",\"answer\":\"The review focuses on immunotherapies, molecularly targeted therapies, and radioligands, highlighting progress beyond conventional chemotherapy.\"},{\"question\":\"What therapeutic areas and drug classes are discussed as examples of immunotherapy in rare pediatric cancers?\",\"answer\":\"Examples include immune checkpoint inhibitors and adoptive cell therapies, including blinatumomab, tisagenlecleucel, and dinutuximab, each transforming treatment in specific pediatric cancers.\"},{\"question\":\"What major challenges limit progress in rare pediatric cancer clinical trials?\",\"answer\":\"The review identifies limited patient numbers for efficient, well-powered trials and a lack of financial incentives, requiring ongoing coordinated efforts to improve survival and minimize late effects.\"}]","Rare but not forgotten - Therapeutic advancements for rare childhood cancers | PDF",1790195401,33]