Treatment of newly diagnosed severe aplastic anemia in children: Evidence-based recommendations.
aplastic anemia
bone marrow failure
pediatric hematology/oncology
Journal
Pediatric blood & cancer
ISSN: 1545-5017
Titre abrégé: Pediatr Blood Cancer
Pays: United States
ID NLM: 101186624
Informations de publication
Date de publication:
16 May 2024
16 May 2024
Historique:
revised:
27
04
2024
received:
12
01
2024
accepted:
29
04
2024
medline:
17
5
2024
pubmed:
17
5
2024
entrez:
17
5
2024
Statut:
aheadofprint
Résumé
Severe aplastic anemia (SAA) is a rare potentially fatal hematologic disorder. Although overall outcomes with treatment are excellent, there are variations in management approach, including differences in treatment between adult and pediatric patients. Certain aspects of treatment are under active investigation in clinical trials. Because of the rarity of the disease, some pediatric hematologists may have relatively limited experience with the complex management of SAA. The following recommendations reflect an up-to-date evidence-based approach to the treatment of children with newly diagnosed SAA.
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
e31070Informations de copyright
© 2024 The Authors. Pediatric Blood & Cancer published by Wiley Periodicals LLC.
Références
Camitta BM, Thomas ED, Nathan DG, et al. Severe aplastic anemia: a prospective study of the effect of early marrow transplantation on acute mortality. Blood. 1976;48(1):63‐70.
Shimano KA, Narla A, Rose MJ, et al. Diagnostic work‐up for severe aplastic anemia in children: Consensus of the North American Pediatric Aplastic Anemia Consortium. Am J Hematol. 2021;96(11):1491‐1504.
Rovo A, Tichelli A, Dufour C, Saa‐Wp E. Diagnosis of acquired aplastic anemia. Bone Marrow Transplant. 2013;48(2):162‐167.
Camitta BM, Storb R, Thomas ED. Aplastic anemia (first of two parts): pathogenesis, diagnosis, treatment, and prognosis. N Engl J Med. 1982;306(11):645‐652.
Li FP, Alter BP, Nathan DG. The mortality of acquired aplastic anemia in children. Blood. 1972;40(2):153‐162.
Camitta BM, Storb R, Thomas ED. Aplastic anemia (second of two parts): pathogenesis, diagnosis, treatment, and prognosis. N Engl J Med. 1982;306(12):712‐718.
Frickhofen N, Rosenfeld SJ. Immunosuppressive treatment of aplastic anemia with antithymocyte globulin and cyclosporine. Semin Hematol. 2000;37(1):56‐68.
Williams DA, Bennett C, Bertuch A, et al. Diagnosis and treatment of pediatric acquired aplastic anemia (AAA): an initial survey of the North American Pediatric Aplastic Anemia Consortium (NAPAAC). Pediatr Blood Cancer. 2014;61(5):869‐874.
Barone A, Lucarelli A, Onofrillo D, et al. Diagnosis and management of acquired aplastic anemia in childhood. Guidelines from the Marrow Failure Study Group of the Pediatric Haemato‐Oncology Italian Association (AIEOP). Blood Cells Mol Dis. 2015;55(1):40‐47.
Samarasinghe S, Veys P, Vora A, Wynn R. Paediatric amendment to adult BSH Guidelines for aplastic anaemia. Br J Haematol. 2018;180(2):201‐205.
Killick SB, Bown N, Cavenagh J, et al. Guidelines for the diagnosis and management of adult aplastic anaemia. Br J Haematol. 2016;172(2):187‐207.
Young NS. Aplastic Anemia. N Engl J Med. 2018;379(17):1643‐1656.
Guyatt G, Oxman AD, Akl EA, et al. GRADE guidelines: 1. Introduction‐GRADE evidence profiles and summary of findings tables. J Clin Epidemiol. 2011;64(4):383‐394.
Guyatt GH, Oxman AD, Kunz R, et al. Going from evidence to recommendations. BMJ. 2008;336(7652):1049‐1051.
Yoshida N, Kobayashi R, Yabe H, et al. First‐line treatment for severe aplastic anemia in children: bone marrow transplantation from a matched family donor versus immunosuppressive therapy. Haematologica. 2014;99(12):1784‐1791.
Dufour C, Pillon M, Socie G, et al. Outcome of aplastic anaemia in children. A study by the severe aplastic anaemia and paediatric disease working parties of the European group blood and bone marrow transplant. Br J Haematol. 2015;169(4):565‐573.
Dufour C, Pillon M, Passweg J, et al. Outcome of aplastic anemia in adolescence: a survey of the Severe Aplastic Anemia Working Party of the European Group for Blood and Marrow Transplantation. Haematologica. 2014;99(10):1574‐1581.
Scheinberg P, Young NS. How I treat acquired aplastic anemia. Blood. 2012;120(6):1185‐1196.
Bacigalupo A. How I treat acquired aplastic anemia. Blood. 2017;129(11):1428‐1436.
Alotaibi H, Aljurf M, de Latour R, et al. Upfront alternative donor transplant versus immunosuppressive therapy in patients with severe aplastic anemia who lack a fully HLA‐matched related donor: systematic review and meta‐analysis of retrospective studies, on behalf of the severe aplastic anemia working party of the european group for blood and marrow transplantation. Transplant Cell Ther. 2022;28(2):105.e101‐105.e107.
Cheng Y, Xu Z, Zhang Y, et al. First‐line choice for severe aplastic anemia in children: Transplantation from a haploidentical donor vs immunosuppressive therapy. Clin Transplant. 2018;32(2).
Choi YB, Yi ES, Lee JW, Sung KW, Koo HH, Yoo KH. Immunosuppressive therapy versus alternative donor hematopoietic stem cell transplantation for children with severe aplastic anemia who lack an HLA‐matched familial donor. Bone Marrow Transplant. 2017;52(1):47‐52.
Xu ZL, Zhou M, Jia JS, et al. Immunosuppressive therapy versus haploidentical transplantation in adults with acquired severe aplastic anemia. Bone Marrow Transplant. 2019;54(8):1319‐1326.
Yang S, Yuan X, Ma R, et al. Comparison of outcomes of frontline immunosuppressive therapy and frontline haploidentical hematopoietic stem cell transplantation for children with severe aplastic anemia who lack an HLA‐matched sibling donor. Biol Blood Marrow Transplant. 2019;25(5):975‐980.
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.
DeZern AE, Zahurak M, Symons HJ, et al. Alternative donor BMT with posttransplant cyclophosphamide as initial therapy for acquired severe aplastic anemia. Blood. 2023;141(25):3031‐3038.
Pulsipher MA, Lehmann LE, Bertuch AA, et al. A study assessing the feasibility of randomization of pediatric and young adult patients between matched unrelated donor bone marrow transplantation and immune‐suppressive therapy for newly diagnosed severe aplastic anemia: A joint pilot trial of the North American Pediatric Aplastic Anemia Consortium and the Pediatric Transplantation and Cellular Therapy Consortium. Pediatr Blood Cancer. 2020;67(10):e28444.
Bacigalupo A, Socie G, Hamladji RM, et al. Current outcome of HLA identical sibling versus unrelated donor transplants in severe aplastic anemia: an EBMT analysis. Haematologica. 2015;100(5):696‐702.
Storb R, Etzioni R, Anasetti C, et al. Cyclophosphamide combined with antithymocyte globulin in preparation for allogeneic marrow transplants in patients with aplastic anemia. Blood. 1994;84(3):941‐949.
Bejanyan N, Kim S, Hebert KM, et al. Choice of conditioning regimens for bone marrow transplantation in severe aplastic anemia. Blood Adv. 2019;3(20):3123‐3131.
Takpradit C, Prockop SE, Kernan NA, et al. Allogeneic matched related donor bone marrow transplantation for pediatric patients with severe aplastic anemia using “low‐dose” cyclophosphamide, ATG plus fludarabine. J Pediatr Hematol Oncol. 2018;40(4):e220‐e224.
Marsh JC, Pearce RM, Koh MB, et al. Retrospective study of alemtuzumab vs ATG‐based conditioning without irradiation for unrelated and matched sibling donor transplants in acquired severe aplastic anemia: a study from the British Society for Blood and Marrow Transplantation. Bone Marrow Transplant. 2014;49(1):42‐48.
Eapen M, Le Rademacher J, Antin JH, et al. Effect of stem cell source on outcomes after unrelated donor transplantation in severe aplastic anemia. Blood. 2011;118(9):2618‐2621.
Bacigalupo A, Socie G, Schrezenmeier H, et al. Bone marrow versus peripheral blood as the stem cell source for sibling transplants in acquired aplastic anemia: survival advantage for bone marrow in all age groups. Haematologica. 2012;97(8):1142‐1148.
Schrezenmeier H, Passweg JR, Marsh JC, et al. Worse outcome and more chronic GVHD with peripheral blood progenitor cells than bone marrow in HLA‐matched sibling donor transplants for young patients with severe acquired aplastic anemia. Blood. 2007;110(4):1397‐1400.
Pagliuca S, Peffault de Latour R, Volt F, et al. Long‐term outcomes of cord blood transplantation from an HLA‐identical sibling for patients with bone marrow failure syndromes: a report from eurocord, cord blood committee and severe aplastic anemia working party of the european society for blood and marrow transplantation. Biol Blood Marrow Transplant. 2017;23(11):1939‐1948.
Stepensky P, Revel‐Vilk S, Weintraub M, et al. Combination of umbilical cord blood with BM from a 2‐month‐old sibling as lifesaving BMT for very severe aplastic anemia. Bone Marrow Transplant. 2008;42(8):563‐564.
Eapen M, Zhang MJ, Tang XY, et al. Hematopoietic cell transplantation with cryopreserved grafts for severe aplastic anemia. Biol Blood Marrow Transplant. 2020;26(7):e161‐e166.
Amare M, Abdou NL, Robinson MG, Abdou NI. Aplastic anemia associated with bone marrow suppressor T‐cell hyperactivity: successful treatment with antithymocyte globulin. Am J Hematol. 1978;5(1):25‐32.
Pedersen‐Bjergaard J, Ernst P, Nissen NI. Severe aplastic anaemia with complete autologous marrow reconstitution following treatment with antithymocyte globulin. Report of a case and review of the literature. Scand J Haematol. 1978;21(1):14‐18.
Cairo MS, Baehner RL. The use of antithymocyte globulin in the treatment of severe aplastic anemia in children. J Pediatr. 1982;100(2):307‐311.
Champlin R, Ho W, Gale RP. Antithymocyte globulin treatment in patients with aplastic anemia: a prospective randomized trial. N Engl J Med. 1983;308(3):113‐118.
Scheinberg P, Fischer SH, Li L, et al. Distinct EBV and CMV reactivation patterns following antibody‐based immunosuppressive regimens in patients with severe aplastic anemia. Blood. 2007;109(8):3219‐3224.
Feng X, Scheinberg P, Biancotto A, et al. In vivo effects of horse and rabbit antithymocyte globulin in patients with severe aplastic anemia. Haematologica. 2014;99(9):1433‐1440.
Lopez M, Clarkson MR, Albin M, Sayegh MH, Najafian N. A novel mechanism of action for anti‐thymocyte globulin: induction of CD4+CD25+Foxp3+ regulatory T cells. J Am Soc Nephrol. 2006;17(10):2844‐2853.
Feng X, Kajigaya S, Solomou EE, et al. Rabbit ATG but not horse ATG promotes expansion of functional CD4+CD25highFOXP3+ regulatory T cells in vitro. Blood. 2008;111(7):3675‐3683.
Scheinberg P, Nunez O, Weinstein B, et al. Horse versus rabbit antithymocyte globulin in acquired aplastic anemia. N Engl J Med. 2011;365(5):430‐438.
Marsh JC, Bacigalupo A, Schrezenmeier H, et al. Prospective study of rabbit antithymocyte globulin and cyclosporine for aplastic anemia from the EBMT Severe Aplastic Anaemia Working Party. Blood. 2012;119(23):5391‐5396.
Yoshimi A, Niemeyer CM, Fuhrer MM, Strahm B. Comparison of the efficacy of rabbit and horse antithymocyte globulin for the treatment of severe aplastic anemia in children. Blood. 2013;121(5):860‐861.
Jeong DC, Chung NG, Cho B, et al. Long‐term outcome after immunosuppressive therapy with horse or rabbit antithymocyte globulin and cyclosporine for severe aplastic anemia in children. Haematologica. 2014;99(4):664‐671.
Rosenfeld SJ, Kimball J, Vining D, Young NS. Intensive immunosuppression with antithymocyte globulin and cyclosporine as treatment for severe acquired aplastic anemia. Blood. 1995;85(11):3058‐3065.
Rogers ZR, Nakano TA, Olson TS, et al. Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study. Haematologica. 2019;104(10):1974‐1983.
Rosenfeld S, Follmann D, Nunez O, Young NS. Antithymocyte globulin and cyclosporine for severe aplastic anemia: association between hematologic response and long‐term outcome. JAMA. 2003;289(9):1130‐1135.
Frickhofen N, Kaltwasser JP. Immunosuppressive treatment of aplastic anemia: a prospective, randomized multicenter trial evaluating antilymphocyte globulin (ALG) versus ALG and cyclosporin A. Blut. 1988;56(4):191‐192.
Frickhofen N, Kaltwasser JP, Schrezenmeier H, et al. Treatment of aplastic anemia with antilymphocyte globulin and methylprednisolone with or without cyclosporine. The German Aplastic Anemia Study Group. N Engl J Med. 1991;324(19):1297‐1304.
Frickhofen N, Heimpel H, Kaltwasser JP, Schrezenmeier H, German Aplastic Anemia Study G. Antithymocyte globulin with or without cyclosporin A: 11‐year follow‐up of a randomized trial comparing treatments of aplastic anemia. Blood. 2003;101(4):1236‐1242.
Marsh J, Schrezenmeier H, Marin P, et al. Prospective randomized multicenter study comparing cyclosporin alone versus the combination of antithymocyte globulin and cyclosporin for treatment of patients with nonsevere aplastic anemia: a report from the European Blood and Marrow Transplant (EBMT) Severe Aplastic Anaemia Working Party. Blood. 1999;93(7):2191‐2195.
Fuhrer M, Burdach S, Ebell W, et al. Relapse and clonal disease in children with aplastic anemia (AA) after immunosuppressive therapy (IST): the SAA 94 experience. German/Austrian Pediatric Aplastic Anemia Working Group. Klin Padiatr. 1998;210(4):173‐179.
Morris RG. Cyclosporin therapeutic drug monitoring–an established service revisited. Clin Biochem Rev. 2003;24(2):33‐46.
Saracco P, Quarello P, Iori AP, et al. Cyclosporin A response and dependence in children with acquired aplastic anaemia: a multicentre retrospective study with long‐term observation follow‐up. Br J Haematol. 2008;140(2):197‐205.
Ratanatharathorn V, Nash RA, Przepiorka D, et al. Phase III study comparing methotrexate and tacrolimus (prograf, FK506) with methotrexate and cyclosporine for graft‐versus‐host disease prophylaxis after HLA‐identical sibling bone marrow transplantation. Blood. 1998;92(7):2303‐2314.
Nash RA, Antin JH, Karanes C, et al. Phase 3 study comparing methotrexate and tacrolimus with methotrexate and cyclosporine for prophylaxis of acute graft‐versus‐host disease after marrow transplantation from unrelated donors. Blood. 2000;96(6):2062‐2068.
Haddad EM, McAlister VC, Renouf E, Malthaner R, Kjaer MS, Gluud LL. Cyclosporin versus tacrolimus for liver transplanted patients. Cochrane Database Syst Rev. 2006;2006(4):CD005161.
Trompeter R, Filler G, Webb NJ, et al. Randomized trial of tacrolimus versus cyclosporin microemulsion in renal transplantation. Pediatr Nephrol. 2002;17(3):141‐149.
Taylor DO, Barr ML, Radovancevic B, et al. A randomized, multicenter comparison of tacrolimus and cyclosporine immunosuppressive regimens in cardiac transplantation: decreased hyperlipidemia and hypertension with tacrolimus. J Heart Lung Transplant. 1999;18(4):336‐345.
Alsultan A, Goldenberg NA, Kaiser N, Graham DK, Hays T. Tacrolimus as an alternative to cyclosporine in the maintenance phase of immunosuppressive therapy for severe aplastic anemia in children. Pediatr Blood Cancer. 2009;52(5):626‐630.
Zhu X, Guan J, Xu J, et al. Pilot study using tacrolimus rather than cyclosporine plus antithymocyte globulin as an immunosuppressive therapy regimen option for severe aplastic anemia in adults. Blood Cells Mol Dis. 2014;53(3):157‐160.
Niu H, Qi W, Wang Y, et al. Successful sirolimus therapy of an aplastic anemia patient with chronic kidney disease: A case report. Medicine (Baltimore). 2020;99(23):e20669.
Scheinberg P, Wu CO, Nunez O, et al. Treatment of severe aplastic anemia with a combination of horse antithymocyte globulin and cyclosporine, with or without sirolimus: a prospective randomized study. Haematologica. 2009;94(3):348‐354.
Millar MM, Grammer LC. Case reports of evaluation and desensitization for anti‐thymocyte globulin hypersensitivity. Ann Allergy Asthma Immunol. 2000;85(4):311‐316.
Wolanin SA, Demain JG, Meier EA. Successful desensitization of a patient with aplastic anemia to antithymocyte globulin. Allergy Rhinol (Providence). 2015;6(1):64‐67.
Chao YK, Shyur SD, Wu CY, Wang CY. Childhood serum sickness: a case report. J Microbiol Immunol Infect. 2001;34(3):220‐223.
Lundquist AL, Chari RS, Wood JH, et al. Serum sickness following rabbit antithymocyte‐globulin induction in a liver transplant recipient: case report and literature review. Liver Transpl. 2007;13(5):647‐650.
Teng J, Hoo XN, Tan SJ, Dwyer K. Serum sickness following rabbit anti‐thymocyte globulin for acute vascular renal allograft rejection. Clin Kidney J. 2012;5(4):334‐335.
Jie M, Fu L, Li S, et al. Efficacy and safety of eltrombopag in the first‐line therapy of severe aplastic anemia in children. Pediatr Hematol Oncol. 2021;38(7):647‐657.
Bacigalupo A, Bruno B, Saracco P, et al. Antilymphocyte globulin, cyclosporine, prednisolone, and granulocyte colony‐stimulating factor for severe aplastic anemia: an update of the GITMO/EBMT study on 100 patients. European Group for Blood and Marrow Transplantation (EBMT) Working Party on Severe Aplastic Anemia and the Gruppo Italiano Trapianti di Midolio Osseo (GITMO). Blood. 2000;95(6):1931‐1934.
Dincol G, Aktan M, Diz‐Kucukkaya R, et al. Treatment of acquired severe aplastic anemia with antilymphocyte globulin, cyclosporin A, methyprednisolone, and granulocyte colony‐stimulating factor. Am J Hematol. 2007;82(9):783‐786.
Boddu P, Garcia‐Manero G, Ravandi F, et al. Clinical outcomes in adult patients with aplastic anemia: A single institution experience. Am J Hematol. 2017;92(12):1295‐1302.
Townsley DM, Desmond R, Dunbar CE, Young NS. Pathophysiology and management of thrombocytopenia in bone marrow failure: possible clinical applications of TPO receptor agonists in aplastic anemia and myelodysplastic syndromes. Int J Hematol. 2013;98(1):48‐55.
Olnes MJ, Scheinberg P, Calvo KR, et al. Eltrombopag and improved hematopoiesis in refractory aplastic anemia. N Engl J Med. 2012;367(1):11‐19.
Kimura S, Roberts AW, Metcalf D, Alexander WS. Hematopoietic stem cell deficiencies in mice lacking c‐Mpl, the receptor for thrombopoietin. Proc Natl Acad Sci USA. 1998;95(3):1195‐1200.
Zeigler FC, de Sauvage F, Widmer HR, et al. In vitro megakaryocytopoietic and thrombopoietic activity of c‐mpl ligand (TPO) on purified murine hematopoietic stem cells. Blood. 1994;84(12):4045‐4052.
Qian H, Buza‐Vidas N, Hyland CD, et al. Critical role of thrombopoietin in maintaining adult quiescent hematopoietic stem cells. Cell Stem Cell. 2007;1(6):671‐684.
FDA‐Label‐Search. Accessed November 30, 2023. https://nctr‐crs.fda.gov/fdalabel/services/spl/set‐ids/7714a0ed‐34bb‐46e6‐a0a5‐b363908b22c2/spl‐doc?hl=eltrombopag
Townsley DM, Scheinberg P, Winkler T, et al. Eltrombopag added to standard immunosuppression for aplastic anemia. N Engl J Med. 2017;376(16):1540‐1550.
Scheinberg P. A new standard immunosuppression regimen in severe aplastic anemia. N Engl J Med. 2022;386(1):89‐90.
Peffault de Latour R, Kulasekararaj A, Iacobelli S, et al. Eltrombopag added to immunosuppression in severe aplastic anemia. N Engl J Med. 2022;386(1):11‐23.
Groarke EM, Patel BA, Gutierrez‐Rodrigues F, et al. Eltrombopag added to immunosuppression for children with treatment‐naive severe aplastic anaemia. Br J Haematol. 2021;192(3):605‐614.
Zaimoku Y, Patel BA, Shalhoub R, et al. Predicting response of severe aplastic anemia to immunosuppression combined with eltrombopag. Haematologica. 2022;107(1):126‐133.
Shimamura A, Maschan A, Bennett C, et al. Eltrombopag in pediatric patients with previously untreated or refractory/relapsed severe aplastic anemia: the Phase II escalate trial. Blood. 2022;140(Supplement 1):705‐707.
Goronkova O, Novichkova G, Salimova T, et al. Efficacy of combined immunosuppression with or without eltrombopag in children with newly diagnosed aplastic anemia. Blood Adv. 2023;7(6):953‐962.
Tichelli A, Schrezenmeier H, Socie G, et al. A randomized controlled study in patients with newly diagnosed severe aplastic anemia receiving antithymocyte globulin (ATG), cyclosporine, with or without G‐CSF: a study of the SAA Working Party of the European Group for Blood and Marrow Transplantation. Blood. 2011;117(17):4434‐4441.
Tichelli A, de Latour RP, Passweg J, et al. Long‐term outcome of a randomized controlled study in patients with newly diagnosed severe aplastic anemia treated with antithymocyte globulin and cyclosporine, with or without granulocyte colony‐stimulating factor: a Severe Aplastic Anemia Working Party Trial from the European Group of Blood and Marrow Transplantation. Haematologica. 2020;105(5):1223‐1231.
Liu X, Zou Y, Wang S, et al. Immunosuppressive therapy using antithymocyte globulin and cyclosporin A with or without human granulocyte colony‐stimulating factor in children with acquired severe aplastic anemia. Zhonghua Er Ke Za Zhi. 2014;52(2):84‐89.
Camitta BM. What is the definition of cure for aplastic anemia? Acta Haematol. 2000;103(1):16‐18.
Hillmen P, Muus P, Duhrsen U, et al. Effect of the complement inhibitor eculizumab on thromboembolism in patients with paroxysmal nocturnal hemoglobinuria. Blood. 2007;110(12):4123‐4128.
Hillmen P, Elebute M, Kelly R, et al. Long‐term effect of the complement inhibitor eculizumab on kidney function in patients with paroxysmal nocturnal hemoglobinuria. Am J Hematol. 2010;85(8):553‐559.
Kelly RJ, Hill A, Arnold LM, et al. Long‐term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival. Blood. 2011;117(25):6786‐6792.
Zhao X, Zhang L, Jing L, et al. The role of paroxysmal nocturnal hemoglobinuria clones in response to immunosuppressive therapy of patients with severe aplastic anemia. Ann Hematol. 2015;94(7):1105‐1110.
Kulagin A, Lisukov I, Ivanova M, et al. Prognostic value of paroxysmal nocturnal haemoglobinuria clone presence in aplastic anaemia patients treated with combined immunosuppression: results of two‐centre prospective study. Br J Haematol. 2014;164(4):546‐554.
Peffault de Latour R, Schrezenmeier H, Bacigalupo A, et al. Allogeneic stem cell transplantation in paroxysmal nocturnal hemoglobinuria. Haematologica. 2012;97(11):1666‐1673.
Fattizzo B, Ireland R, Dunlop A, et al. Clinical and prognostic significance of small paroxysmal nocturnal hemoglobinuria clones in myelodysplastic syndrome and aplastic anemia. Leukemia. 2021;35(11):3223‐3231.
Stanley N, Olson TS, Babushok DV. Recent advances in understanding clonal haematopoiesis in aplastic anaemia. Br J Haematol. 2017;177(4):509‐525.
Babushok DV. When does a PNH clone have clinical significance? Hematol Am Soc Hematol Educ Program. 2021;2021(1):143‐152.
Sabet K, Panigrahi AR. Pediatric patients with severe aplastic anemia treated with combined immunosuppressive therapy, eltrombopag and eculizumab and their clinical course to stem cell transplant. Blood. 2020;136(Supplement 1):13‐13.
Alashkar F, Duhrsen U, Roth A. Horse anti‐thymocyte globulin and eculizumab as concomitant therapeutic approach in an aplastic paroxysmal nocturnal hemoglobinuria patient: go or no‐go? Eur J Haematol. 2016;97(4):403‐405.
Pagliuca S, Risitano AM, De Fontbrune FS, et al. Combined intensive immunosuppression and eculizumab for aplastic anemia in the context of hemolytic paroxysmal nocturnal hemoglobinuria: a retrospective analysis. Bone Marrow Transplant. 2018;53(1):105‐107.
Kulasekararaj AG, Hill A, Rottinghaus ST, et al. Ravulizumab (ALXN1210) vs eculizumab in C5‐inhibitor‐experienced adult patients with PNH: the 302 study. Blood. 2019;133(6):540‐549.
Roth A, Nishimura JI, Nagy Z, et al. The complement C5 inhibitor crovalimab in paroxysmal nocturnal hemoglobinuria. Blood. 2020;135(12):912‐920.
Hillmen P, Szer J, Weitz I, et al. Pegcetacoplan versus eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2021;384(11):1028‐1037.
Risitano AM, Peffault de Latour R. How we('ll) treat paroxysmal nocturnal haemoglobinuria: diving into the future. Br J Haematol. 2021;196(2):288‐303.