Special issues related to the diagnosis and management of acquired aplastic anemia in countries with restricted resources, a report on behalf of the Eastern Mediterranean blood and marrow transplantation (EMBMT) group and severe aplastic anemia working party of the European Society for blood and marrow transplantation (SAAWP of EBMT).

Anemia, Aplastic / diagnosis Bone Marrow Bone Marrow Failure Disorders Bone Marrow Transplantation Hematopoietic Stem Cell Transplantation Humans Transplantation Conditioning

Journal

Bone marrow transplantation

ISSN: 1476-5365

Titre abrégé: Bone Marrow Transplant

Pays: England

ID NLM: 8702459

Informations de publication

Date de publication:
10 2021

Historique:

received: 09 03 2021

accepted: 26 04 2021

revised: 30 03 2021

pubmed: 21 5 2021

medline: 14 10 2021

entrez: 20 5 2021

Statut: ppublish

Résumé

Aplastic anemia is a relatively rare but potentially fatal disorder, with a reported higher incidence in developing countries in comparison to the West. There are significant variations in epidemiological as well as etiological factors of bone marrow failure syndromes in the developing countries in comparison to the developed world. Furthermore, the management of bone marrow failure syndromes in resource constraint settings has significant challenges including delayed diagnosis and referral, limited accessibility to healthcare facilities, treatment modalities as well as limitations related to patients who require allogeneic stem cell transplantation. Here we will provide a review of the available evidence related to specific issues of aplastic anemia in the developing countries and we summarize suggested recommendations from the Eastern Mediterranean blood and bone marrow transplantation (EMBMT) group and the severe aplastic anemia working party of the European Society of blood and marrow transplantation (SAAWP of EBMT) related to the diagnosis and therapeutic options in countries with restricted resources.

Identifiants

DOI: 10.1038/s41409-021-01332-8 PMID: 34011966

pubmed: 34011966

doi: 10.1038/s41409-021-01332-8

pii: 10.1038/s41409-021-01332-8

doi:

Types de publication

Journal Article Review

Langues

eng

Sous-ensembles de citation

Pagination

2518-2532

Investigateurs

Tarek Ben Othman (T)

Regis de Latour (R)

Commentaires et corrections

Type : ErratumIn

Informations de copyright

Références

Killick SB, Bown N, Cavenagh J, Dokal I, Foukaneli T, Hill A, et al. Guidelines for the diagnosis and management of adult aplastic anaemia. Br J Haematol. 2016;172:187–207.

pubmed: 26568159 doi: 10.1111/bjh.13853

Kojima S. Why is the incidence of aplastic anemia higher in Asia? Expert Rev Hematol. 2017;10:277–9.

pubmed: 28264622 doi: 10.1080/17474086.2017.1302797

Ally M, Magesa P, Luzzatto L. High frequency of acquired aplastic anemia in Tanzania. Am J Hematol. 2019;94:E86–8.

pubmed: 30592075 doi: 10.1002/ajh.25388

Peslak SA, Olson T, Babushok DV. Diagnosis and treatment of aplastic anemia. Curr Treat Options Oncol. 2017;18:70.

pubmed: 29143887 pmcid: 5804354 doi: 10.1007/s11864-017-0511-z

Camitta BM, Storb R, Thomas ED. Aplastic anemia (first of two parts): pathogenesis, diagnosis, treatment, and prognosis. N Engl J Med. 1982;306:645–52.

pubmed: 7035946 doi: 10.1056/NEJM198203183061105

Savage SA, Viard M, O’HUigin C, Zhou W, Yeager M, Li SA, et al. Genome-wide Association Study Identifies HLA-DPB1 as a significant risk factor for severe aplastic anemia. Am J Hum Genet. 2020;106:264–71.

pubmed: 32004448 pmcid: 7010969 doi: 10.1016/j.ajhg.2020.01.004

Dolberg OJ, Levy Y. Idiopathic aplastic anemia: diagnosis and classification. Autoimmun Rev. 2014;13:569–73.

pubmed: 24424170 doi: 10.1016/j.autrev.2014.01.014

Gadalla SM, Savage SA. Telomere biology in hematopoiesis and stem cell transplantation. Blood Rev. 2011;25:261–9.

pubmed: 21764192 pmcid: 7737640 doi: 10.1016/j.blre.2011.06.004

Young NS, Kaufman DW. The epidemiology of acquired aplastic anemia. Haematologica. 2008;93:489–92.

pubmed: 18379007 doi: 10.3324/haematol.12855

Montané E, Ibáñez L, Vidal X, Ballarín E, Puig R, García N, et al. Epidemiology of aplastic anemia: a prospective multicenter study. Haematologica. 2008;93:518–23.

pubmed: 18322256 doi: 10.3324/haematol.12020

Ahmed P, Chaudhry QUN, Satti TM, Mahmood SK, Ghafoor T, Shahbaz N, et al. Epidemiology of aplastic anemia: a study of 1324 cases. Hematology. 2020;25:48–54.

pubmed: 31906834 doi: 10.1080/16078454.2019.1711344

Akram Z, Ahmed P, Kajigaya S, Satti TM, Satti HS, Chaudhary QUN, et al. Epidemiological, clinical and genetic characterization of aplastic anemia patients in Pakistan. Ann Hematol. 2019;98:301–12.

pubmed: 30426156 doi: 10.1007/s00277-018-3542-z

Rehman S, Saba N, Naz M, Ahmed P, Munir S, Sajjad S, et al. Single-nucleotide polymorphisms of FAS and FASL genes and risk of idiopathic aplastic anemia. Immunol Investig. 2018;47:484–91.

doi: 10.1080/08820139.2018.1458106

Rehman S, Ahmed P, Saba N, Munir S, Sajjad S, Satti TM, et al. Association of GSTM1 and GSTT1 deletion polymorphisms with Pakistani aplastic anemia patients and controls and meta-analysis. Ann Hematol. 2015;94:1965–71.

pubmed: 26327568 doi: 10.1007/s00277-015-2482-0

Mahapatra M, Singh PK, Agarwal M, Prabhu M, Mishra P, Seth T, et al. Epidemiology, clinico-haematological profile and management of aplastic anaemia: AIIMS experience. J Assoc Physicians India. 2015;63 (3 Suppl):30–5.

pubmed: 26529865

Prihartono N, Kriebel D, Woskie S, Thetkhathuek A, Sripaung N, Padungtod C, et al. Risk of aplastic anemia and pesticide and other chemical exposures. Asia Pac J Public Health. 2011;23:369–77.

pubmed: 21490113 doi: 10.1177/1010539511403605

Young NS, Issaragrasil S, Chieh CW, Takaku F. Aplastic anaemia in the Orient. Br J Haematol. 1986;62:1–6.

pubmed: 3942690 doi: 10.1111/j.1365-2141.1986.tb02893.x

Wang L, Liu H. Pathogenesis of aplastic anemia. Hematology. 2019;24:559–66.

pubmed: 31315542 doi: 10.1080/16078454.2019.1642548

Mary JY, Baumelou E, Guiguet M. Epidemiology of aplastic anemia in France: a prospective multicentric study. The French Cooperative Group for Epidemiological Study of Aplastic Anemia. Blood. 1990;75:1646–53.

Shallis RM, Ahmad R, Zeidan AM. Aplastic anemia: Etiology, molecular pathogenesis, and emerging concepts. Eur J Haematol. 2018;101:711–20.

pubmed: 30055055 doi: 10.1111/ejh.13153

Issaragrisil S, Kaufman DW, Anderson T, Chansung K, Leaverton PE, Shapiro S, et al. The epidemiology of aplastic anemia in Thailand. Blood. 2006;107:1299–307.

pubmed: 16254144 pmcid: 1895423 doi: 10.1182/blood-2005-01-0161

Yong AS, Goh AS, Rahman J, Menon J, Purushothaman V. Epidemiology of aplastic anemia in the state of Sabah, Malaysia. Med J Malaysia. 1998;53:59–62.

pubmed: 10968139

Ahamed M, Anand M, Kumar A, Siddiqui MKJ. Childhood aplastic anaemia in Lucknow, India: incidence, organochlorines in the blood and review of case reports following exposure to pesticides. Clin Biochem. 2006;39:762–6.

pubmed: 16729992 doi: 10.1016/j.clinbiochem.2006.03.021

Jeong DC, Chung NG, Kang HJ, Koo HH, Kook H, Kim SK, et al. Epidemiology and clinical long-term outcome of childhood aplastic anemia in Korea for 15 years: retrospective study of the Korean Society of Pediatric Hematology Oncology (KSPHO). J Pediatr Hematol Oncol. 2011;33:172–8.

pubmed: 21325968 doi: 10.1097/MPH.0b013e31820826a8

Arewa OP, Akinola NO. Survival in primary a plastic anaemia; experience with 20 cases from a tertiary hospital in Nigeria. Afr Health Sci. 2009;9:290–3.

pubmed: 21503184 pmcid: 3074392

Adil S, Kakepoto GN, Khurshid M, Burney IA. Epidemiological features of aplastic anaemia in Pakistan. J Pak Med Assoc. 2001;51:443.

pubmed: 11850982

Ehrlich P. Ueber einem Fall von Anämie mit Bemerkungen über regenerative Veränderungen des Knochenmarks. Charité-Annalen. 1888;13:301–9.

Smith MT. Overview of benzene-induced aplastic anaemia. Eur J Haematol. 1996;60:107–10.

Aksoy M. Benzene as a leukemogenic and carcinogenic agent. Am J Ind Med. 1985;8:9–20.

pubmed: 4025342 doi: 10.1002/ajim.4700080103

Cudillo L. Aplastica anemia and viral hepatitis. Mediterr J Hematol Infect Dis. 2009;1:e2009026.

pubmed: 21415960 pmcid: 3033128

Alter BP. Aplastic anemia, pediatric aspects. Oncologist. 1996;1:361–6.

pubmed: 10388017 doi: 10.1634/theoncologist.1-6-361

McCahon E, Tang K, Rogers PC, McBride ML, Schultz KR. The impact of Asian descent on the incidence of acquired severe aplastic anaemia in children. Br J Haematol. 2003;121:170–2.

pubmed: 12670349 doi: 10.1046/j.1365-2141.2003.04236.x

Chen C-H, Ferreira JC, Joshi AU, Stevens MC, Li S-J, Hsu JH-M, et al. Novel and prevalent non-East Asian ALDH2 variants; implications for global susceptibility to aldehydes’ toxicity. EBioMedicine. 2020;55:102753.

pubmed: 32403082 pmcid: 7218264 doi: 10.1016/j.ebiom.2020.102753

Rehman S, Saba N, Khalilullah, Munir S, Ahmed P, Mehmood T. The frequency of HLA class I and II alleles in Pakistani patients with aplastic anemia. Immunol Investig. 2009;38:812–9.

doi: 10.3109/08820130903271415

Lee KA, Kim SH, Woo HY, Hong YJ, Cho HC. Increased frequencies of glutathione S-transferase (GSTM1 and GSTT1) gene deletions in Korean patients with acquired aplastic anemia. Blood. 2001;98:3483–5.

pubmed: 11719393 doi: 10.1182/blood.V98.12.3483

Sleijfer S, Lugtenburg P. Aplastic anaemia: a review. Neth J Med. 2003;61:157–63.

pubmed: 12916541

Dutta A, De R, Dolai TK, Mitra PK, Halder A. Comparative study of environmental factors and different addictions in aplastic anaemia patients in Eastern India. J Blood Disord Symptoms Treat. 2019;8:2015–8.

Taj M, Shah T, Aslam SK, Zaheer S, Nawab F, Shaheen S, et al. Environmental determinants of aplastic anemia in Pakistan: a case-control study. J Public Health. 2016;24:453–60.

pubmed: 27695669 doi: 10.1007/s10389-016-0743-6

Marsh JCW. Aplastic anemia: what’s in the environment? Blood. 2006;107:1250.

doi: 10.1182/blood-2005-11-4652

Akram Z, Ahmed P, Kajigaya S, Satti TM, Satti HS, Gutierrez-Rodrigues F, et al. Epidemiological, clinical and genetic characterization of aplastic anemia patients in Pakistan. Ann Hematol. 2019;98:301–12.

pubmed: 30426156 doi: 10.1007/s00277-018-3542-z

Kook H, Chung NG, Kang HJ, Im HJ. Acquired aplastic anemia in Korean children: treatment guidelines from the Bone Marrow Failure Committee of the Korean Society of Pediatric Hematology Oncology. Int J Hematol. 2016;103:380–6.

pubmed: 26894608 doi: 10.1007/s12185-016-1956-8

Ramzan M, Yadav SP, Zafar MS, Dinand V, Sachdeva A. Outcome of pediatric acquired aplastic anemia: a developing world experience. Pediatr Hematol Oncol. 2014;31:29–38.

pubmed: 23802844 doi: 10.3109/08880018.2013.807898

Townsley DM, Dumitriu B, Young NS. Bone marrow failure and the telomeropathies. Blood. 2014;124:2775–83.

pubmed: 25237198 pmcid: 4215309 doi: 10.1182/blood-2014-05-526285

Alter BP. Inherited bone marrow failure syndromes: considerations pre- and posttransplant. Blood. 2017;130:2257–64.

pubmed: 29167174 pmcid: 5714231 doi: 10.1182/blood-2017-05-781799

Narayanan S. Managing anaemia in bone marrow failure syndromes. Curr Opin Support Palliat Care. 2018;12:538–41.

pubmed: 30299326 doi: 10.1097/SPC.0000000000000397

Young NS. Current concepts in the pathophysiology and treatment of aplastic anemia. Hematol Am Soc Hematol Educ Program. 2013;2013:76–81.

doi: 10.1182/asheducation-2013.1.76

Höchsmann B, Moicean A, Risitano A, Ljungman P, Schrezenmeier H. Supportive care in severe and very severe aplastic anemia. Bone Marrow Transplant. 2013;48:168–73.

pubmed: 23208312 doi: 10.1038/bmt.2012.220

Han W. Health care system reforms in developing countries. J Public Health Res. 2012;1:199.

pubmed: 25170464 pmcid: 4140377 doi: 10.4081/jphr.2012.e31

Valdez JM, Scheinberg P, Young NS, Walsh TJ. Infections in patients with aplastic anemia. Semin Hematol. 2009;46:269–76.

pubmed: 19549579 doi: 10.1053/j.seminhematol.2009.03.008

Young NS. Aplastic Anemia. N Engl J Med. 2018;379:1643–56.

pubmed: 30354958 pmcid: 6467577 doi: 10.1056/NEJMra1413485

Zhang Y, Wu L, Mo W, Zhou M, Li Y, Chen X, et al. Comparable outcomes of first-line hematopoietic stem cell transplantation from unrelated and matched sibling donors in adult patients with aplastic anemia: a retrospective single-center study. Biol Blood Marrow Transplant. 2019;25:1567–75.

pubmed: 30926448 doi: 10.1016/j.bbmt.2019.03.020

Marsh JC, Risitano AM, Mufti GJ. The case for upfront HLA-matched unrelated donor hematopoietic stem cell transplantation as a curative option for adult acquired severe aplastic anemia. Biol Blood Marrow Transplant. 2019;25:e277–84.

pubmed: 31129354 doi: 10.1016/j.bbmt.2019.05.012

Xu LP, Jin S, Wang SQ, Xia LH, Bai H, Gao SJ, et al. Upfront haploidentical transplant for acquired severe aplastic anemia: registry-based comparison with matched related transplant. J Hematol Oncol. 2017;10:25.

pubmed: 28107815 pmcid: 5251320 doi: 10.1186/s13045-017-0398-y

Chaudhry QUN, Iftikhar R, Satti TM, Mahmood SK, Ghafoor T, Shamshad GU, et al. Outcome of fludarabine-based conditioning in high-risk aplastic anemia patients undergoing matched related donor transplantation: a single-center study from Pakistan. Biol Blood Marrow Transplant. 2019;25:2375–82.

pubmed: 31394274 doi: 10.1016/j.bbmt.2019.07.029

Jubelirer SJ. The benefit of the neutropenic diet: fact or fiction? Oncologist. 2011;16:704.

pubmed: 21471277 pmcid: 3228185 doi: 10.1634/theoncologist.2011-0001

Quillen K, Wong E, Scheinberg P, Young NS, Walsh TJ, Wu CO, et al. Granulocyte transfusions in severe aplastic anemia: an eleven-year experience. Haematologica. 2009;94:1661–8.

pubmed: 19996117 pmcid: 2791947 doi: 10.3324/haematol.2009.010231

Garg A, Gupta A, Mishra A, Singh M, Yadav S, Nityanand S. Role of granulocyte transfusions in combating life-threatening infections in patients with severe neutropenia: experience from a tertiary care centre in North India. PloS ONE. 2018;13:e0209832.

pubmed: 30589898 pmcid: 6307785 doi: 10.1371/journal.pone.0209832

Kallen ME, Dulau-Florea A, Wang W, Calvo KR. Acquired and germline predisposition to bone marrow failure: Diagnostic features and clinical implications. Semin Hematol. 2019;56:69–82.

pubmed: 30573048 doi: 10.1053/j.seminhematol.2018.05.016

Townsley DM, Scheinberg P, Winkler T, Desmond R, Dumitriu B, Rios O, et al. Eltrombopag added to standard immunosuppression for aplastic anemia. N Engl J Med. 2017;376:1540–50.

pubmed: 28423296 pmcid: 5548296 doi: 10.1056/NEJMoa1613878

Cle DV, Atta EH, Dias DSP, Lima CBL, Bonduel M, Sciuccati G, et al. Rabbit antithymocyte globulin dose does not affect response or survival as first-line therapy for acquired aplastic anemia: a multicenter retrospective study. Ann Hematol. 2018;97:2039–46.

pubmed: 29978284 doi: 10.1007/s00277-018-3416-4

Lum SH, Grainger JD. Eltrombopag for the treatment of aplastic anemia: current perspectives. Drug Des Devel Ther. 2016;10:2833–43.

pubmed: 27695288 pmcid: 5028092 doi: 10.2147/DDDT.S95715

Al-Ghazaly J, Al-Dubai W, Al-Jahafi AK, Abdullah M, Al-Hashdi A. Cyclosporine monotherapy for severe aplastic anemia: a developing country experience. Ann Saudi Med. 2005;25:375–9.

pubmed: 16270758 pmcid: 6089711 doi: 10.5144/0256-4947.2005.375

Maschan A, Bogatcheva N, Kryjanovskii O, Shneider M, Litvinov D, Mitiushkina T, et al. Results at a single centre of immunosuppression with cyclosporine A in 66 children with aplastic anaemia. Br J Haematol. 1999;106:967–70.

pubmed: 10519999 doi: 10.1046/j.1365-2141.1999.01650.x

Mandal PK, Baul S, Dolai TK, De R, Chakrabarti P. Outcome of cyclosporine monotherapy in patients of aplastic anemia: experience of a Tertiary Care Hospital in Eastern India. Indian J Hematol Blood Transfus. 2017;33:144–7.

pubmed: 28194076 doi: 10.1007/s12288-016-0706-7

Scheinberg P, Nunez O, Weinstein B, Scheinberg P, Biancotto A, Wu CO, et al. Horse versus rabbit antithymocyte globulin in acquired aplastic anemia. N Engl J Med. 2011;365:430–8.

pubmed: 21812672 pmcid: 3721503 doi: 10.1056/NEJMoa1103975

Marsh JC, Bacigalupo A, Schrezenmeier H, Tichelli A, Risitano AM, Passweg JR, et al. Prospective study of rabbit antithymocyte globulin and cyclosporine for aplastic anemia from the EBMT Severe Aplastic Anaemia Working Party. Blood. 2012;119:5391–6.

pubmed: 22544699 doi: 10.1182/blood-2012-02-407684

Risitano AM, Perna F. Aplastic anemia: immunosuppressive therapy in 2010. Pediatr Rep. 2011;3:14–7.

doi: 10.4081/pr.2011.s2.e7

Feng X, Scheinberg P, Biancotto A, Rios O, Donaldson S, Wu C, et al. In vivo effects of horse and rabbit antithymocyte globulin in patients with severe aplastic anemia. Haematologica. 2014;99:1433–40.

pubmed: 24907357 pmcid: 4562531 doi: 10.3324/haematol.2014.106542

Scheinberg P, Wu CO, Nunez O, Young NS. Predicting response to immunosuppressive therapy and survival in severe aplastic anaemia. Br J Haematol. 2009;144:206–16.

pubmed: 19036108 doi: 10.1111/j.1365-2141.2008.07450.x

Lengline E, Drenou B, Peterlin P, Tournilhac O, Abraham J, Berceanu A, et al. Nationwide survey on the use of eltrombopag in patients with severe aplastic anemia: a report on behalf of the French Reference Center for Aplastic Anemia. Haematologica. 2018;103:212–20.

pubmed: 29170252 pmcid: 5792265 doi: 10.3324/haematol.2017.176339

Ecsedi M, Lengline É, Knol-Bout C, Bosman P, Eikema D-J, Afanasyev B, et al. Use of eltrombopag in aplastic anemia in Europe. Ann Hematol. 2019;98:1341–50.

pubmed: 30915499 doi: 10.1007/s00277-019-03652-8

Konishi A, Nakaya A, Fujita S, Satake A, Nakanishi T, Azuma Y, et al. Evaluation of eltrombopag in patients with aplastic anemia in real-world experience. Leuk Res Rep. 2019;11:11–3.

pubmed: 30911464 pmcid: 6416522

Zhu Y, Gao Q, Hu J, Liu X, Guan D, Zhang F. Allo-HSCT compared with immunosuppressive therapy for acquired aplastic anemia: a system review and meta-analysis. BMC Immunol. 2020;21:10.

pubmed: 32138642 pmcid: 7059290 doi: 10.1186/s12865-020-0340-x

Dufour C, Pillon M, Sociè G, Rovò A, Carraro E, Bacigalupo A, 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;169:565–73.

pubmed: 25683884 doi: 10.1111/bjh.13297

Dufour C, Pillon M, Passweg J, Socie G, Bacigalupo A, Franceschetto G, 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;99:1574–81.

pubmed: 25085353 pmcid: 4181253 doi: 10.3324/haematol.2014.106096

Georges GE. Consider allogeneic bone marrow transplantation for older, fitpatients with aplastic anemia. Biol Blood Marrow Transplant. 2019;25:e69–70.

pubmed: 30641134 doi: 10.1016/j.bbmt.2019.01.015

Sheth VS, Potter V, Gandhi SA, Kulasekararaj AG, de Lavallade H, Muus P, et al. Similar outcomes of alemtuzumab-based hematopoietic cell transplantation for SAA patients older or younger than 50 years. Blood Adv. 2019;3:3070–9.

pubmed: 31648330 pmcid: 6849949 doi: 10.1182/bloodadvances.2019000480

Zhang YY, Mo WJ, Zuo YY, Zhou M, Zhang XH, Wang Y, et al. Comparable survival outcome between transplantation from haploidentical donor and matched related donor or unrelated donor for severe aplastic anemia patients aged 40 years and older: a retrospective multicenter cohort study. Clin Transplant. 2020;34:e13810.

pubmed: 32011059 doi: 10.1111/ctr.13810

Geng C, Liu X, Chen M, Yang C, Han B. Comparison of frontline treatment with intensive immunosuppression therapy and HLA-haploidentical hematopoietic stem cell transplantation for young patients with severe aplastic anemia—a meta analysis. Leuk Res. 2020;88:106266.

pubmed: 31743865 doi: 10.1016/j.leukres.2019.106266

Prata PH, Eikema DJ, Afansyev B, Bosman P, Smiers F, Diez-Martin JL, et al. Haploidentical transplantation and posttransplant cyclophosphamide for treating aplastic anemia patients: a report from the EBMT Severe Aplastic Anemia Working Party. Bone Marrow Transplant. 2020;55:1050–58.

pubmed: 31844137 doi: 10.1038/s41409-019-0773-0

Ciceri F, Lupo-Stanghellini M, Korthof E. Haploidentical transplantation in patients with acquired aplastic anemia. Bone Marrow Transplant. 2013;48:183–5.

pubmed: 23292235 doi: 10.1038/bmt.2012.231

Luo CJ, Chen J, Wang JM, Qin X, Zhang BH, Zhu H, et al. Alternative donor HSCT for 109 children with acquired severe aplastic anemia: a single center retrospective analysis. Zhonghua Xue Ye Xue Za Zhi. 2020;41:128–31.

pubmed: 32135629

Yoshida N, Kojima S. Updated guidelines for the treatment of acquired aplastic anemia in children. Curr Oncol Rep. 2018;20:67.

pubmed: 29961134 doi: 10.1007/s11912-018-0716-8

Dufour C, Veys P, Carraro E, Bhatnagar N, Pillon M, Wynn R, 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;171:585–94.

pubmed: 26223288 doi: 10.1111/bjh.13614

Yue C, Ding Y, Gao Y, Li L, Pang Y, Liu Z, et al. Cotransplantation of haploidentical hematopoietic stem cells and allogeneic bone marrow-derived mesenchymal stromal cells as a first-line treatment in very severe aplastic anemia patients with refractory infections. Eur J Haematol. 2018;100:624–9.

pubmed: 29532539 doi: 10.1111/ejh.13060

Lu Y, Sun RJ, Zhao YL, Xiong M, Cao XY, Zhang JP, et al. Unmanipulated haploidentical hematopoietic stem cell transplantation achieved outcomes comparable with matched unrelated donor transplantation in young acquired severe aplastic anemia. Biol Blood Marrow Transplant. 2018;24:1881–7.

pubmed: 29772350 doi: 10.1016/j.bbmt.2018.05.015

Li Y, Duan F, Xiao H, Wu X, Wang S, Xu D, et al. Therapeutic outcomes of haploidentical allogeneic hematopoietic stem cell transplantation in patients with severe aplastic anemia: a multicenter study. Transplantation. 2018;102:1724–31.

pubmed: 29677078 doi: 10.1097/TP.0000000000002200

Kim H, Lee KH, Sohn SK, Kim I, Kim SH, Park Y, et al. Effect of stem cell source and dose on allogeneic hematopoietic stem cell transplantation in adult patients with idiopathic aplastic anemia: data from the korean aplastic anemia trials. Acta Haematol. 2020;143:232–43.

pubmed: 31390612 doi: 10.1159/000501496

Bacigalupo A, Socie G, Schrezenmeier H, Tichelli A, Locasciulli A, Fuehrer M, 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;97:1142–8.

pubmed: 22315497 pmcid: 3409810 doi: 10.3324/haematol.2011.054841

Schrezenmeier H, Passweg JR, Marsh JC, Bacigalupo A, Bredeson CN, Bullorsky E, 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;110:1397–400.

pubmed: 17475907 pmcid: 1939910 doi: 10.1182/blood-2007-03-081596

Kumar R, Kimura F, Ahn KW, Hu Z-H, Kuwatsuka Y, Klein JP, et al. Comparing outcomes with bone marrow or peripheral blood stem cells as graft source for matched sibling transplants in severe aplastic anemia across different economic regions. Biol Blood Marrow Transplant. 2016;22:932–40.

pubmed: 26797402 pmcid: 4826285 doi: 10.1016/j.bbmt.2016.01.012

Morton J, Hutchins C, Durrant S. Granulocyte–colony-stimulating factor (G-CSF)–primed allogeneic bone marrow: significantly less graft-versus-host disease and comparable engraftment to G-CSF–mobilized peripheral blood stem cells. Blood. 2001;98:3186–91.

pubmed: 11719353 doi: 10.1182/blood.V98.12.3186

Clay J, Kulasekararaj AG, Potter V, Grimaldi F, McLornan D, Raj K, et al. Nonmyeloablative peripheral blood haploidentical stem cell transplantation for refractory severe aplastic anemia. Biol Blood Marrow Transplant. 2014;20:1711–6.

pubmed: 25016195 doi: 10.1016/j.bbmt.2014.06.028

Storb R, Etzioni R, Anasetti C, Appelbaum FR, Buckner CD, Bensinger W, et al. Cyclophosphamide combined with antithymocyte globulin in preparation for allogeneic marrow transplants in patients with aplastic anemia. Blood. 1994;84:941–9.

pubmed: 8043876 doi: 10.1182/blood.V84.3.941.941

Aljurf M, Al-Zahrani H, Van Lint M, Passweg J. Standard treatment of acquired SAA in adult patients 18–40 years old with an HLA-identical sibling donor. Bone Marrow Transplant. 2013;48:178–9.

pubmed: 23165494 doi: 10.1038/bmt.2012.223

Iftikhar R, Anwer F, Neupane K, Rafae A, Mahmood SK, Ghafoor T, et al. Allogeneic hematopoietic stem cell transplantation in aplastic anemia: current indications and transplant strategies. Blood Rev. 2020;47:100772.

pubmed: 33187812 doi: 10.1016/j.blre.2020.100772

Iftikhar R, Satti TM, Mahmood SK, Ghafoor T, Shamshad GU, Shahbaz N, et al. Comparison of Conventional Cyclophosphamide versus Fludarabine-Based Conditioning in High-Risk Aplastic Anemia Patients Undergoing Matched-Related Donor Transplantation. Clin Hematol Int. 2020;2:82–91.

pubmed: 34595447 pmcid: 8432348 doi: 10.2991/chi.d.200426.001

Aljurf M, Zaidi SZ, El Solh H, Hussain F, Ghavamzadeh A, Mahmoud HK, et al. Special issues related to hematopoietic SCT in the Eastern Mediterranean region and the first regional activity report. Bone Marrow Transplant. 2009;43:1–12.

pubmed: 19043456 doi: 10.1038/bmt.2008.389

Chen JY, Zheng TL, Zhou T, Hu PW, Huang MJ, Xu X, et al. Human cytomegalovirus prevalence and distribution of glycoprotein B, O genotypes among hospitalized children with respiratory infections in West China, 2009–2014. Trop Med Int Health. 2016;21:1428–34.

pubmed: 27515771 doi: 10.1111/tmi.12770

Tomblyn M, Chiller T, Einsele H, Gress R, Sepkowitz K, Storek J, et al. Guidelines for preventing infectious complications among hematopoietic cell transplant recipients: a global perspective. Bone Marrow Transplant. 2009;44:453–5.

pubmed: 19861977 doi: 10.1038/bmt.2009.254

Burroughs LM, Woolfrey AE, Storer BE, Deeg HJ, Flowers ME, Martin PJ, et al. Success of allogeneic marrow transplantation for children with severe aplastic anaemia. Br J Haematol. 2012;158:120–8.

pubmed: 22533862 pmcid: 3693852 doi: 10.1111/j.1365-2141.2012.09130.x

Marsh JC, Gupta V, Lim Z, Ho AY, Ireland RM, Hayden J, et al. Alemtuzumab with fludarabine and cyclophosphamide reduces chronic graft-versus-host disease after allogeneic stem cell transplantation for acquired aplastic anemia. Blood. 2011;118:2351–7.

pubmed: 21518925 doi: 10.1182/blood-2010-12-327536

Vaht K, Goransson M, Carlson K, Isaksson C, Lenhoff S, Sandstedt A, et al. High graft-versus-host disease-free, relapse/rejection-free survival and similar outcome of related and unrelated allogeneic stem cell transplantation for aplastic anemia: a Nationwide Swedish Cohort Study. Biol Blood Marrow Transplant. 2019;25:1970–4.

pubmed: 31173901 doi: 10.1016/j.bbmt.2019.05.032

George B, Mathews V, Viswabandya A, Kavitha ML, Srivastava A, Chandy M. Fludarabine and cyclophosphamide based reduced intensity conditioning (RIC) regimens reduce rejection and improve outcome in Indian patients undergoing allogeneic stem cell transplantation for severe aplastic anemia. Bone Marrow Transplant. 2007;40:13–8.

pubmed: 17450183 doi: 10.1038/sj.bmt.1705669

Jalili M, Alimoghaddam K, Hamidieh AA, Hamdi A, Jahani M, Bahar B, et al. Hematopoietic stem cell transplantation in patients with severe acquired aplastic anemia: Iranian experience. Int J Hematol-Oncol Stem Cell Res. 2011;5:22–7.

Atta EH, de Sousa AM, Schirmer MR, Bouzas LF, Nucci M, Abdelhay E. Different outcomes between cyclophosphamide plus horse or rabbit antithymocyte globulin for HLA-identical sibling bone marrow transplant in severe aplastic anemia. Biol Blood Marrow Transplant. 2012;18:1876–82.

pubmed: 22796534 doi: 10.1016/j.bbmt.2012.07.004

Shin SH, Lee JW. The optimal immunosuppressive therapy for aplastic anemia. Int J Hematol. 2013;97:564–72.

pubmed: 23605367 doi: 10.1007/s12185-013-1331-y

Agarwal M, Jijina F, Shah S, Malhotra P, Damodar S, Ross C. Safety and efficacy of indigenous equine antithymocyte globulin along with cyclosporine in subjects with acquired aplastic anemia. Indian J Hematol Blood Transfus. 2015;31:174–9.

pubmed: 25825555 doi: 10.1007/s12288-014-0423-z

Zheng Y, Liu Y, Chu Y. Immunosuppressive therapy for acquired severe aplastic anemia (SAA): a prospective comparison of four different regimens. Exp Hematol. 2006;34:826–31.

pubmed: 16797409 doi: 10.1016/j.exphem.2006.03.017