Wskazania do transplantacji komórek krwiotwórczych u dzieci i młodzieży w 2024 roku — rekomendacje Polskiej Pediatrycznej Grupy ds. Transplantacji Komórek Krwiotwórczych
Streszczenie
U dzieci i młodzieży transplantacja komórek krwiotwórczych (HSCT, hematopoietic stem cell transplantation) znajduje zastosowanie w chorobach nowotworowych oraz we wrodzonych i nabytych chorobach nieonkologicznych. Wskazania do HSCT ustalane są w kontekście aktualnych wyników leczenia z zastosowaniem innych opcji terapeutycznych — HSCT jest wskazana tylko wtedy, gdy jest jedyną skuteczną metodą leczenia lub gdy znamiennie zwiększa szanse chorego na wyleczenie. Ze względu na postępy leczenia konwencjonalnego oraz immunoterapii, terapii ukierunkowanej molekularnie i terapii genowej, a także z uwagi na postępy w zakresie procedury HSCT, wskazania do transplantacji muszą być regularnie aktualizowane. Dlatego Polska Pediatryczna Grupa ds. Transplantacji Komórek Krwiotwórczych Polskiego Towarzystwa Onkologii i Hematologii Dziecięcej (PTOHD) po raz kolejny nowelizuje rekomendacje dotyczące wskazań do HSCT w populacji pediatrycznej z uwzględnieniem bieżących zaleceń European Society for Blood and Bone Marrow Transplantation (EBMT), w tym zaleceń EBMT Pediatric Diseases Working Party oraz EBMT Inborn Errors Working Party, a także wytycznych zawartych w protokołach terapeutycznych aktualnie obowiązujących w 18 ośrodkach onkologii i hematologii dziecięcej akredytowanych przez PTOHD. Przedstawione rekomendacje skierowane są w pierwszym rzędzie do ośrodków onkologii i hematologii dziecięcej oraz do ośrodków, w których prowadzona jest diagnostyka i leczenie pierwotnych niedoborów odporności i wrodzonych chorób metabolicznych, bowiem to na tych ośrodkach spoczywa obowiązek zidentyfikowania wskazań do HSCT i skierowania pacjenta w odpowiednim czasie do transplantacji w jednym z sześciu dziecięcych ośrodków przeszczepiania komórek krwiotwórczych w Polsce. Ośrodki te posiadają obecnie ogółem 45 stanowisk transplantacyjnych, co gwarantuje przeprowadzenie HSCT w optymalnym punkcie czasowym terapii u każdego pacjenta pediatrycznego ze wskazaniami do transplantacji.
Słowa kluczowe: transplantacja komórek krwiotwórczychwskazaniadziecimłodzież
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