Wyszuk. zaawans.

  • Hematologia — Edukacja
  • Tom 5, Nr 1-2 (2024) Wskazania do transplantacji komórek krwiotwórczych u dzieci i młodzieży w 2024 roku — rekomendacje Polskiej Pediatrycznej Grupy ds. Transplantacji Komórek Krwiotwórczych
Tom 5, Nr 1-2 (2024)
Wytyczne / stanowisko ekspertów
Opublikowany online: 2024-10-01

dostęp otwarty

Pokaż PDF Pobierz plik PDF
Wyświetlenia strony 169
Wyświetlenia/pobrania artykułu 139
Pobierz cytowanie

Eksport do Mediów Społecznościowych

Eksport do Mediów Społecznościowych

Wskazania do transplantacji komórek krwiotwórczych u dzieci i młodzieży w 2024 roku — rekomendacje Polskiej Pediatrycznej Grupy ds. Transplantacji Komórek Krwiotwórczych

Jacek Wachowiak1, Krzysztof Czyżewski2, Katarzyna Drabko3, Jolanta Goździk4, Bernarda Kazanowska5, Iwona Malinowska6, Monika Mielcarek-Siedziuk5, Anna Pieczonka1, Agnieszka Sobkowiak-Sobierajska1, Marek Ussowicz5, Grażyna Wróbel5, Krzysztof Kałwak5, Jan Styczyński2
DOI: 10.5603/hemedu.100852
Hematologia — Edukacja 2024;5(1-2):1-17.

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. Aktualnie ośrodki te posiadają 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

Artykuł dostępny w formacie PDF

Pokaż PDF Pobierz plik PDF

Referencje

  1. AIEOP-BFM ALL 2017 POLAND — Wspólny protokół leczenia dzieci i młodzieży z ostrą białaczką limfoblastyczną. Randomizowane badanie III fazy prowadzone w porozumieniu z grupą badawczą AIEOP-BFM, wersja 2.0 z dn. 3.12.2020 r.
  2. Peters C, Dalle JH, Locatelli F, et al. Total body irradiation or chemotherapy conditioning in childhood ALL: a multinational, randomized, noninferiority phase III study. J Clin Oncol. 2021; 39(4): 295–307.
    CrossRefPubMed
  3. International phase 3 trial in Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) testing imatinib in combination with two different cytotoxic chemotherapy backbones — EsPhALL2017/COGAALL1631,EsPhALL network I-BFM Study Group Children’s Oncology Group. Version 3.1 z dnia 15.01.2021 r.
  4. IntReALL 2010 HR — high risk group — international study for treatment of childhood relapsed ALL 2010. Protocol Version 2. 2, Date. ; 21: July.
  5. IntReALL 2010 SR — standard risk — international study for treatment of standard risk childhood relapsed ALL 2010. Protocol Version 1.91, Date 12.08.2016.
  6. Locatelli F, Zugmaier G, Rizzari C, et al. Effect of blinatumomab vs chemotherapy on event-free survival among children with high-risk first-relapse B-cell acute lymphoblastic leukemia: a randomized clinical trial. JAMA. 2021; 325(9): 843–854.
    CrossRefPubMed
  7. Program lekowy B.65 Leczenie chorych na ostrą białaczkę limfoblastyczną.
  8. Recommendations for diagnostics, therapy, and follow-up care of children and adolescents with acute myeloid leukemia — AML-BFM Study Group version 12/2023.
  9. International multicenter, open-label clinical trial for the treatment of acute myeloid leukemia in children and adolescents (AIEOP-BFM-AML 2020).
  10. Abla O, Kutny MA, Testi AM, et al. Management of relapsed and refractory childhood acute promyelocytic leukemia: recommendations from internationl expert panel. Br J Haematol. 2016; 175(4): 588–601.
    CrossRefPubMed
  11. Waack K, Röllecke K, Rasche M, et al. Treatment‐Related acute myeloid leukemia in children. Blood. 2019; 134(Suppl_1): 1322–1322.
    CrossRef
  12. Czogała M, Czogała W, Pawińska-Wąsikowska K, et al. Pediatric acute myeloid leukemia post cytotoxic therapy-retrospective analysis of the patients treated in Poland from 2005 to 2022. Cancers (Basel). 2023; 15(3).
    CrossRefPubMed
  13. Hrusak O, de Haas V, Stancikova J, et al. International cooperative study identifies treatment strategy in childhood ambiguous lineage leukemia. Blood. 2018; 132(3): 264–276.
    CrossRefPubMed
  14. Lee HG, Baek HJo, Kim HoS, et al. Biphenotypic acute leukemia or acute leukemia of ambiguous lineage in childhood: clinical characteristics and outcome. Blood Res. 2019; 54(1): 63–73.
    CrossRefPubMed
  15. CML-paed-II — protocol for standarized diagnostic procedures, registration, and treatment recommendations in children and adolescents with Philadelphia chromosome-positive chronić myeloid leukemia (CML). Version: November 2006.
  16. De la Fuente J, Baruchel A, Biondi A, et al. Managing children with chronic myeloid leukaemia (CML) Recommendations for the management of CML in children and young people up to the age of 18 years. Br J Haematol. 2014; 167(1): 33–47.
    CrossRefPubMed
  17. Hijiya N, Schultz KR, Metzler M, et al. Pediatric chronic myeloid leukemia is a unique disease that requires a different approach. Blood. 2016; 127(4): 392–399.
    CrossRefPubMed
  18. Sembill S, Ampatzidou M, Chaudhury S, et al. Management of children and adolescents with chronic myeloid leukemia in blast phase: International pediatric CML expert panel recommendations. Leukemia. 2023; 37(3): 505–517.
    CrossRefPubMed
  19. Pichler H, Sedlacek P, Meisel R, et al. Haematopoietic stem cell transplantation after reduced intensity conditioning in children and adolescents with chronic myeloid leukaemia: A prospective multicentre trial of the I-BFM Study Group. Br J Haematol. 2024; 205(1): 268–279.
    CrossRefPubMed
  20. Guidelines for Hematopoietic Stem Cell Transplantation (HSCT) in Childhood MDS and JMML for Patients enrolled in EWOG-MDS Studies. https://ewog-mds.de/fileadmin/mediapool/10_andere/ewog-mds/pdf/protocoldocs/other/ConsensusGuidelinesHSCTinMDSandJMML_v1.34.pdf (23.03.2024).
  21. EWOG-MDS 2006. Prospective non–randomized multi-center study for epidemiology and characterization of Myelodysplastic Syndromes (MDS) and Juvenile Myelomonocytic Leukemia (JMML) in childhood. https://ewog-mds.de/fileadmin/mediapool/10_andere/ewog-mds/pdf/protocoldocs/mds_2006/masterprotocol20061130international.pdf (23.03.2024).
  22. Sahoo SS, Pastor VB, Goodings C, et al. European Working Group of MDS in Children (EWOG-MDS), European Working Group of MDS in Children (EWOG-MDS). Clinical evolution, genetic landscape and trajectories of clonal hematopoiesis in SAMD9/SAMD9L syndromes. Nat Med. 2021; 27(10): 1806–1817.
    CrossRefPubMed
  23. Arber DA, Orazi A, Hasserjian RP, et al. International Consensus Classification of Myeloid Neoplasms and Acute Leukemias: integrating morphologic, clinical, and genomic data. Blood. 2022; 140(11): 1200–1228.
    CrossRefPubMed
  24. Kucine N. Myeloproliferative neoplasms in children, adolescents, and young adults. Curr Hematol Malig Rep. 2020; 15(2): 141–148.
    CrossRefPubMed
  25. Kröger N, McLornan DP, Chalandon Y. Myeloproliferative neoplasms. In: Sureda A, Corbacioglu S, Greco R. et al. ed. EBMT handbook – hematopoietic stem cell transplantation and cellular therapies. Springer, Cham 2024: 695–705.
  26. McLornan D, Eikema DJ, Czerw T, et al. Trends in allogeneic haematopoietic cell transplantation for myelofibrosis in Europe between 1995 and 2018: a CMWP of EBMT retrospective analysis. Bone Marrow Transplant. 2021; 56(9): 2160–2172.
    CrossRefPubMed
  27. Wachowiak J, Galimard JE, Dalissier A, et al. Outcomes of allogeneic haematopoietic cell transplantation for myelofibrosis in children and adolescents: the retrospective study of the EBMT Paediatric Diseases WP. Bone Marrow Transplant. 2024; 59(8): 1057–1069.
    CrossRefPubMed
  28. Murthy GS, Kim S, Estrada-Merly N, et al. Association between the choice of the conditioning regimen and outcomes of allogeneic hematopoietic cell transplantation for myelofibrosis. Haematologica. 2023; 108(7): 1900–1908.
    CrossRefPubMed
  29. Kröger N, Sbianchi G, Sirait T, et al. Impact of prior JAK-inhibitor therapy with ruxolitinib on outcome after allogeneic hematopoietic stem cell transplantation for myelofibrosis: a study of the CMWP of EBMT. Leukemia. 2021; 35(12): 3551–3560.
    CrossRefPubMed
  30. Malato A, Rossi E, Tiribelli M, et al. Splenectomy in Myelofibrosis: Indications, Efficacy, and Complications. Clin Lymphoma Myeloma Leuk. 2020; 20(9): 588–595.
    CrossRefPubMed
  31. Beishuizen A, Mellgren K, Andrés M, et al. European Intergroup for Childhood Non-Hodgkin Lymphoma. Improving outcomes of childhood and young adult non-Hodgkin lymphoma: 25 years of research and collaboration within the framework of the European Intergroup for Childhood Non-Hodgkin Lymphoma. Lancet Haematol. 2023; 10(3): e213–e224.
    CrossRefPubMed
  32. Burkhardt B, Taj M, Garnier N, et al. Treatment and Outcome Analysis of 639 Relapsed Non-Hodgkin Lymphomas in Children and Adolescents and Resulting Treatment Recommendations. Cancers (Basel). 2021; 13(9).
    CrossRefPubMed
  33. Ji Qi, Wu X, Zhang Y, et al. Adverse events and efficacy of second-round CAR-T cell therapy in relapsed pediatric B-ALL. Eur J Haematol. 2024; 112(1): 75–82.
    CrossRefPubMed
  34. Temple WC, Mueller S, Hermiston ML, et al. Diagnosis and management of lymphoblastic lymphoma in children, adolescents and young adults. Best Pract Res Clin Haematol. 2023; 36(1): 101449.
    CrossRefPubMed
  35. ALCL-Relapse 2012 – EICNHL treatment protocol for relapsed anaplastic large cell lymphoma of childhood and adolescence. Version 2.0 Last modification 17.09.2012.
  36. Knörr F, Brugières L, Pillon M, et al. European Inter-Group for Childhood Non-Hodgkin Lymphoma. Stem cell transplantation and vinblastine monotherapy for relapsed pediatric anaplastic large cell lymphoma: results of the international, prospective alcl-relapse trial. J Clin Oncol. 2020; 38(34): 3999–4009.
    CrossRefPubMed
  37. Daw S, Hasenclever D, Mascarin M, et al. Risk and Response Adapted Treatment Guidelines for Managing First Relapsed and Refractory Classical Hodgkin Lymphoma in Children and Young People. Recommendations from the EuroNet Pediatric Hodgkin Lymphoma Group. Hemasphere. 2020; 4(1): e329.
    CrossRefPubMed
  38. Testi AM, Moleti ML, Angi A, et al. Pediatric autologous hematopoietic stem cell transplantation: safety, efficacy, and patient outcomes. Literature review. Pediatric Health Med Ther. 2023; 14: 197–215.
    CrossRefPubMed
  39. Massano D, Carraro E, Mussolin L, et al. Brentuximab vedotin in the treatment of paediatric patients with relapsed or refractory Hodgkin's lymphoma: results of a real-life study. Pediatr Blood Cancer. 2022; 69(10): e29801.
    CrossRefPubMed
  40. Pezeshki PS, Eskian M, Hamblin MR, et al. Immune checkpoint inhibition in classical hodgkin lymphoma. Expert Rev Anticancer Ther. 2021; 21(9): 1003–1016.
    CrossRefPubMed
  41. Veys PA, Nanduri V, Baker KS, et al. Haematopoietic stem cell transplantation for refractory Langerhans cell histiocytosis: outcome by intensity of conditioning. Br J Haematol. 2015; 169(5): 711–718.
    CrossRefPubMed
  42. Rodriguez-Galindo C. Clinical features and treatment of Langerhans cell histiocytosis. Acta Paediatr. 2021; 110(11): 2892–2902.
    CrossRefPubMed
  43. Rodriguez-Galindo C, Allen CE. Langerhans cell histiocytosis. Blood. 2020; 135(16): 1319–1331.
    CrossRefPubMed
  44. Ehl S, Astigarraga I, von Bahr Greenwood T, et al. Recommendations for the use of etoposide-based therapy and bone marrow transplantation for the treatment of HLH: consensus statements by the HLH steering committee of the histiocyte society. J Allergy Clin Immunol Pract. 2018; 6(5): 1508–1517.
    CrossRefPubMed
  45. Lankester AC, Albert MH, Booth C, et al. Inborn Errors Working Party of the European Society for Blood and Marrow Transplantation and the European Society for Immune Deficiencies, and European Reference Network on Rare Primary Immunodeficiency Autoinflammatory Autoimmune diseases (RITA). EBMT/ESID inborn errors working party guidelines for hematopoietic stem cell transplantation for inborn errors of immunity. Bone Marrow Transplant. 2021; 56(9): 2052–2062.
    CrossRefPubMed
  46. Ge J, Zhang Q, Ma H, et al. Ruxolitinib-based regimen in children with primary hemophagocytic lymphohistiocytosis. Haematologica. 2024; 109(2): 458–465.
    CrossRefPubMed
  47. Marsh RA, Hebert K, Kim S, et al. Comparison of hematopoietic cell transplant conditioning regimens for hemophagocytic lymphohistiocytosis disorders. J Allergy Clin Immunol. 2022; 149(3): 1097–1104.e2.
    CrossRefPubMed
  48. Snowden JA, Sánchez-Ortega I, Corbacioglu S, et al. European Society for Blood and Marrow Transplantation (EBMT). Indications for haematopoietic cell transplantation for haematological diseases, solid tumours and immune disorders: current practice in Europe, 2022. Bone Marrow Transplant. 2022; 57(8): 1217–1239.
    CrossRefPubMed
  49. Ladenstein R, Pedrazzoli P, Rosti G. Solid tumours. In: Sureda A, Corbacioglu S, Greco R. et al. ed. EBMT Handbook – Hematopoietic Stem Cell Transplantation and Cellular Therapies. Springer, Cham 2024: 849–862.
  50. SIOP Europe. High-risk neuroblastoma standard clinical practice recommendations. https://siope.eu/media/documents/escp-high-risk-neuroblastoma-standard-clinical-practice-recommendations.pdf (20.03.2024).
  51. Albert MH, Lankester A, Genery A. et al. Inborn errors of immunity. In: Sureda A, Corbacioglu S, Greco R. et al. ed. EBMT Handbook – Hematopoietic Stem Cell Transplantation and Cellular Therapies. Springer, Cham 2024: 809–817.
  52. Bernatowska EA, Wolska-Kusnierz B, Pac M, et al. Disseminated bacillus Calmette-Guérin infection and immunodeficiency. Emerg Infect Dis. 2007; 13(5): 799–801.
    CrossRefPubMed
  53. Lankester AC, Albert MH, Booth C, et al. Inborn Errors Working Party of the European Society for Blood and Marrow Transplantation and the European Society for Immune Deficiencies, and European Reference Network on Rare Primary Immunodeficiency Autoinflammatory Autoimmune diseases (RITA). EBMT/ESID inborn errors working party guidelines for hematopoietic stem cell transplantation for inborn errors of immunity. Bone Marrow Transplant. 2021; 56(9): 2052–2062.
    CrossRefPubMed
  54. Chiesa R, Wang J, Blok HJ, et al. Hematopoietic cell transplantation in chronic granulomatous disease: a study of 712 children and adults. Blood. 2020; 136(10): 1201–1211.
    CrossRefPubMed
  55. Kohn LA, Kohn D. Gene therapies for primary immune deficiencies. Front Immunol. 2021; 12: 648951.
    CrossRefPubMed
  56. Ussowicz M, Wawrzyniak-Dzierżek E, Mielcarek-Siedziuk M, et al. Allogeneic stem cell transplantation after fanconi anemia conditioning in children with ataxia-telangiectasia results in stable t cell engraftment and lack of infections despite mixed chimerism. Biol Blood Marrow Transplant. 2018; 24(11): 2245–2249.
    CrossRefPubMed
  57. Bakhtiar S, Fekadu J, Seidel MG, et al. Allogeneic hematopoietic stem cell transplantation for congenital immune dysregulatory disorders. Front Pediatr. 2019; 7: 461.
    CrossRefPubMed
  58. Pefault de Latour R, Risitano A, Kulasekarrraj A. et al. Acquire bone marrow failure: severeaplastic anemia and paroxysmal nocturnal hemoglobinuria. In: Sureda A, Corbacioglu S, Greco R. et al. ed. EBMT Handbook – Hematopoietic Stem Cell Transplantation and Cellular Therapies. Springer, Cham 2024: 707–715.
  59. Locasciulli A, Oneto R, Bacigalupo A, et al. Severe Aplastic Anemia Working Party of the European Blood and Marrow Transplant Group. Outcome of patients with acquired aplastic anemia given first line bone marrow transplantation or immunosuppressive treatment in the last decade: a report from the European Group for Blood and Marrow Transplantation (EBMT). Haematologica. 2007; 92(1): 11–18.
    CrossRefPubMed
  60. Dufour C, Veys P, Carraro E, et al. Similar outcome of upfront-unrelated and matched sibling stem cell transplantation in idiopathic paediatric aplastic anaemia. A study on behalf of the UK Paediatric BMT Working Party, Paediatric Diseases Working Party and Severe Aplastic Anaemia Working Party of EBMT. Br J Haematol. 2015; 171(4): 585–594.
    CrossRefPubMed
  61. EWOG-SAA. Consensus for the treatment of Severe aplastic anaemia in children and adolescents. https://ewog-mds-saa.org/fileadmin/mediapool/10_andere/ewog-mds/pdf/protocoldocs/Concensus_for_treatment_of_SAA_v1.7.pdf (21.03.2024).
  62. MacMillan ML, Wagner JE. Haematopoeitic cell transplantation for Fanconi anaemia — when and how? Br J Haematol. 2010; 149(1): 14–21.
    CrossRefPubMed
  63. Dufour C. How I manage patients with Fanconi anaemia. Br J Haematol. 2017; 178(1): 32–47.
    CrossRefPubMed
  64. Dufour C, Pierri F. Modern management of Fanconi anemia. Hematol Am Soc Hematol Educ Program. 2022; 2022(1): 649–657.
    CrossRefPubMed
  65. Frohnmayer L, Van Ravenhorst S, Wirkkula L. Fanconi Anemia Clinical Care Guidelines, 5th ed. Fanconi Anemia Research Fund, Eugene 2020.
  66. Peffault de Latour R, Peters C, Gibson B, et al. Pediatric Working Party of the European Group for Blood and Marrow Transplantation, Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation. Recommendations on hematopoietic stem cell transplantation for inherited bone marrow failure syndromes. Bone Marrow Transplant. 2015; 50(9): 1168–1172.
    CrossRefPubMed
  67. Diaz-de-Heredia C, Bresters D, Faulkner L, et al. Recommendations on hematopoietic stem cell transplantation for patients with Diamond-Blackfan anemia. On behalf of the Pediatric Diseases and Severe Aplastic Anemia Working Parties of the EBMT. Bone Marrow Transplant. 2021; 56(12): 2956–2963.
    CrossRefPubMed
  68. Rotulo GA, Beaupain B, Rialland F, et al. HSCT may lower leukemia risk in ELANE neutropenia: a before-after study from the French Severe Congenital Neutropenia Registry. Bone Marrow Transplant. 2020; 55(8): 1614–1622.
    CrossRefPubMed
  69. Cesaro S, Donadieu J, Cipolli M, et al. Stem cell transplantation in patients affected by shwachman-diamond syndrome: expert consensus and recommendations from the EBMT severe aplastic anaemia working party. Transplant Cell Ther. 2022; 28(10): 637–649.
    CrossRefPubMed
  70. Fioredda F, Iacobelli S, Korthof ET, et al. Outcome of haematopoietic stem cell transplantation in dyskeratosis congenita. Br J Haematol. 2018; 183(1): 110–118.
    CrossRefPubMed
  71. Wynn R, Schulz A. Inborn errors of metabolism and osteopetrosis. In: Sureda A, Corbacioglu S, Greco R. et al. ed. EBMT Handbook – Hematopoietic Stem Cell Transplantation and Cellular Therapies. Springer, Cham 2024: 819–824.
  72. Boelens JJ, Aldenhoven M, Purtill D, et al. Eurocord, Inborn Errors Working Party of European Blood and Marrow Transplant group, Duke University Blood and Marrow Transplantation Program, Centre for International Blood and Marrow Research. Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning. Blood. 2013; 121(19): 3981–3987.
    CrossRefPubMed
  73. Guffon N, Pettazzoni M, Pangaud N, et al. Long term disease burden post-transplantation: three decades of observations in 25 Hurler patients successfully treated with hematopoietic stem cell transplantation (HSCT). Orphanet J Rare Dis. 2021; 16(1): 60.
    CrossRefPubMed
  74. Armstrong N, Olaye A, Noake C, et al. A systematic review of clinical effectiveness and safety for historical and current treatment options for metachromatic leukodystrophy in children, including atidarsagene autotemcel. Orphanet J Rare Dis. 2023; 18(1): 248.
    CrossRefPubMed
  75. Engelen M, van Ballegoij WJC, Mallack EJ, et al. International recommendations for the diagnosis and management of patients with adrenoleukodystrophy: a consensus-based approach. Neurology. 2022; 99(21): 940–951.
    CrossRefPubMed
  76. Moser AB, Jones RO, Hubbard WC, et al. Newborn screening for X-linked adrenoleukodystrophy. Int J Neonatal Screen. 2016; 2(4).
    CrossRefPubMed
  77. https://www.ebmt.org/ebmt/news/report-inborn-errors-working-party-annual-conference-22-24-september-2023-graz-austria..
  78. Falkowska A, Bachnacka A, Mielcarek-Siedziuk M, et al. Hematopoietic stem cell transplantation in inborn errors of metabolism — Polish experience. Bone Marrow Transplant. 2013; 58(Suppl. 1): 578–630.
  79. Alexander T, Greco R, Snowden JA. Hematopoietic stem cell transplantation for autoimmune disease. Annu Rev Med. 2021; 72: 215–228.
    CrossRefPubMed
  80. Achini-Gutzwiller FR, Snowden JA, Corbacioglu S, et al. EBMT Autoimmune Diseases (ADWP) and Paediatric Diseases (PDWP) Working Parties. Haematopoietic stem cell transplantation for severe autoimmune diseases in children: A review of current literature, registry activity and future directions on behalf of the autoimmune diseases and paediatric diseases working parties of the European Society for Blood and Marrow Transplantation. Br J Haematol. 2022; 198(1): 24–45.
    CrossRefPubMed
  81. Greco R, Alexander T, Burman J, et al. European Society for Blood and Marrow Transplantation (EBMT) Autoimmune Diseases Working Party (ADWP), Infectious Diseases Working Party (IDWP), Pediatric Working Party (PWP), Joint Accreditation Committee of the International Society for Cellular Therapy (ISCT) and EBMT (JACIE), EBMT Nurses Group and Patient Advocacy Committee. Hematopoietic stem cell transplantation for autoimmune diseases in the time of COVID-19: EBMT guidelines and recommendations. Bone Marrow Transplant. 2021; 56(7): 1493–1508.
    CrossRefPubMed
  82. Greco R, Labopin M, Badoglio M, et al. Allogeneic HSCT for Autoimmune Diseases: A Retrospective Study From the EBMT ADWP, IEWP, and PDWP Working Parties. Front Immunol. 2019; 10: 1570.
    CrossRefPubMed
  83. Burman J, Kirgizov K, Carlson K, et al. Autologous hematopoietic stem cell transplantation for pediatric multiple sclerosis: a registry-based study of the Autoimmune Diseases Working Party (ADWP) and Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT). Bone Marrow Transplant. 2017; 52(8): 1133–1137.
    CrossRefPubMed
  84. Barzaghi F, Amaya Hernandez LC, Neven B, et al. Primary Immune Deficiency Treatment Consortium (PIDTC) and the Inborn Errors Working Party (IEWP) of the European Society for Blood and Marrow Transplantation (EBMT). Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study. J Allergy Clin Immunol. 2018; 141(3): 1036–1049.e5.
    CrossRefPubMed
  85. Snowden JA, Panés J, Alexander T, et al. European Crohn’s and Colitis Organisation (ECCO), European Society for Blood and Marrow Transplantation (EBMT), Autoimmune Diseases Working Party (ADWP), Joint Accreditation Committee of the International Society for Cellular Therapy (ISCT) and EBMT (JACIE). Autologous Haematopoietic Stem Cell Transplantation (AHSCT) in Severe Crohn's Disease: A Review on Behalf of ECCO and EBMT. J Crohns Colitis. 2018; 12(4): 476–488.
    CrossRefPubMed
  86. Rabusin M, Snowden JA, Veys P, et al. European Group for Blood and Marrow Transplantation Autoimmune Diseases and Paediatric Disease Working Parties. Long-term outcomes of hematopoietic stem cell transplantation for severe treatment-resistant autoimmune cytopenia in children. Biol Blood Marrow Transplant. 2013; 19(4): 666–669.
    CrossRefPubMed
  87. Silva JMF, Ladomenou F, Carpenter B, et al. Allogeneic hematopoietic stem cell transplantation for severe, refractory juvenile idiopathic arthritis. Blood Adv. 2018; 2(7): 777–786.
    CrossRefPubMed

Informacje o plikach cookie

Niezbędne pliki cookie

Zawsze aktywne

Te pliki cookie są niezbędne do prawidłowego wyświetlania i działania strony internetowej. Zablokowanie ich może spowodować nieprawidłowe działanie strony.

Analityczne pliki cookie

W ramach naszej strony internetowej korzystamy z narzędzi analitycznych, takich jak Google Analytics, które umożliwiają nam weryfikację ruchu na stronie internetowej. Narzędzia te mogą wymagać użycia plików cookie.

Społecznościowe pliki cookie

Są to pliki cookie powiązane z portalami społecznościowymi, z których korzystamy. Zaakceptowanie ich pozwoli na powiązanie Twojej wizyty na stronie z naszymi profilami w mediach społecznościowych.

Marketingowe pliki cookie

Są to pliki cookie odpowiedzialne za prowadzenie działań reklamowych i marketingowych - zgoda na ich wykorzystanie pozwoli nam kierować do Ciebie reklamy, bazujące na podstawie Twoich wcześniejszych aktywności w ramach naszej stronie internetowej.

Copyright © 2023-2024 Via Medica Regulamin Polityka cookies Polityka prywatności Nota prawna