Medulloblastoma in children with Fanconi anemia: Association with FA-D1/FA-N, SHH type and poor survival independent of treatment strategies.
Marthe SönksenDenise ObrechtPablo Hernáiz DrieverAxel SauerbreyNorbert GrafUdo KontnyChristian ReimannMina LangheinUwe R KordesRudolf SchwarzTobias ObserFelix BoschannUlrich SchüllerLea AltendorfTobias GoschzikTorsten PietschMartin MynarekStefan RutkowskiPublished in: Neuro-oncology (2024)
MB in FA patients is strongly associated with SHH activation and FA-D1/FA-N. Despite the dismal prognosis, adjuvant therapy may prolong survival. Non-alkylating chemotherapy and RT are feasible in selected patients with careful monitoring of toxicities and dose adjustments. Curative therapy for FA MB-SHH remains an unmet medical need.