Survival After Hematopoietic Stem Cell Transplantation in Severe Combined Immunodeficiency (SCID): A Worldwide Review of the Prognostic Variables

Tangye SG, Al-Herz W, Bousfiha A, Cunningham-Rundles C, Franco JL, Holland SM et al (2022) Human inborn errors of immunity: 2022 update on the classification from the International Union of Immunological Societies Expert Committee. J Clin Immunol 42(7):1473–1507. https://doi.org/10.1007/s10875-022-01289-3. Epub: 20220624; PubMed PMID: 35748970; PubMed Central PMCID: PMC9244088

Article  PubMed  PubMed Central  Google Scholar 

Dvorak CC, Haddad E, Heimall J, Dunn E, Buckley RH, Kohn DB et al (2023) The diagnosis of severe combined immunodeficiency (SCID): The Primary Immune Deficiency Treatment Consortium (PIDTC) 2022 definitions. J Allergy Clin Immunol 151(2):539–46. https://doi.org/10.1016/j.jaci.2022.10.022. Epub: 20221128; PubMed PMID: 36456361; PubMed Central PMCID: PMC9905311

Article  CAS  PubMed  Google Scholar 

Dvorak CC, Cowan MJ, Logan BR, Notarangelo LD, Griffith LM, Puck JM et al (2013) The natural history of children with severe combined immunodeficiency: baseline features of the first fifty patients of the primary immune deficiency treatment consortium prospective study 6901. J Clin Immunol 33(7):1156–1164. https://doi.org/10.1007/s10875-013-9917-y

Article  CAS  PubMed  PubMed Central  Google Scholar 

Gatti RA, Meuwissen HJ, Allen HD, Hong R, Good RA (1968) Immunological reconstitution of sex-linked lymphopenic immunological deficiency. Lancet 2(7583):1366–1369. https://doi.org/10.1016/s0140-6736(68)92673-1. PubMed PMID: 4177932

Article  CAS  PubMed  Google Scholar 

Gaspar HB, Aiuti A, Porta F, Candotti F, Hershfield MS, Notarangelo LD (2009) How I treat ADA deficiency. Blood 114(17):3524–3532. https://doi.org/10.1182/blood-2009-06-189209. Epub: 20090728; PubMed PMID: 19638621; PubMed Central PMCID: PMC2766674

Article  CAS  PubMed  PubMed Central  Google Scholar 

Fischer A, Hacein-Bey-Abina S, Cavazzana-Calvo M (2011) Gene therapy for primary adaptive immune deficiencies. J Allergy Clin Immunol 127(6):1356–1359. https://doi.org/10.1016/j.jaci.2011.04.030. PubMed PMID: 21624615

Article  CAS  PubMed  Google Scholar 

Hacein-Bey-Abina S, Pai SY, Gaspar HB, Armant M, Berry CC, Blanche S et al (2014) A modified γ-retrovirus vector for X-linked severe combined immunodeficiency. N Engl J Med 371(15):1407–1417. https://doi.org/10.1056/NEJMoa1404588.PubMed PMID: 25295500; PubMed Central PMCID: PMC4274995

Article  PubMed  PubMed Central  Google Scholar 

Kohn DB (2015) Gene therapy outpaces haplo for SCID-X1. Blood 125(23):3521–3522. https://doi.org/10.1182/blood-2015-04-641720. PubMed PMID: 26045591

Article  CAS  PubMed  Google Scholar 

Lankester AC, Albert MH, Booth C, Gennery AR, Güngör T, Hönig M et al (2021) EBMT/ESID inborn errors working party guidelines for hematopoietic stem cell transplantation for inborn errors of immunity. Bone Marrow Transplant 56(9):2052–2062. https://doi.org/10.1038/s41409-021-01378-8. Epub: 20210705; PubMed PMID: 34226669; PubMed Central PMCID: PMC8410590

Article  CAS  PubMed  PubMed Central  Google Scholar 

Roifman CM, Grunebaum E, Dalal I, Notarangelo L (2007) Matched unrelated bone marrow transplant for severe combined immunodeficiency. Immunol Res 38(1–3):191–200. https://doi.org/10.1007/s12026-007-0042-y. PubMed PMID: 17917025

Article  PubMed  Google Scholar 

Dvorak CC, Hassan A, Slatter MA, Hönig M, Lankester AC, Buckley RH et al (2014) Comparison of outcomes of hematopoietic stem cell transplantation without chemotherapy conditioning by using matched sibling and unrelated donors for treatment of severe combined immunodeficiency. Journal of Allergy and Clinical Immunology 134(4):935–43.e15. https://doi.org/10.1016/j.jaci.2014.06.021

Article  PubMed  Google Scholar 

Heimall J, Logan BR, Cowan MJ, Notarangelo LD, Griffith LM, Puck JM et al (2017) Immune reconstitution and survival of 100 SCID patients post-hematopoietic cell transplant: A PIDTC natural history study. Blood 130(25):2718–2727. https://doi.org/10.1182/blood-2017-05-781849

Article  CAS  PubMed  PubMed Central  Google Scholar 

Haddad E, Logan BR, Griffith LM, Buckley RH, Parrott RE, Prockop SE et al (2018) SCID genotype and 6-month posttransplant CD4 count predict survival and immune recovery. Blood 132(17):1737–1749. https://doi.org/10.1182/blood-2018-03-840702

Article  CAS  PubMed  PubMed Central  Google Scholar 

Bayram O, Haskologlu S, Bayrakoğlu D, Bal SK, Islamoglu C, Cipe FE et al (2021) Single-center study of 72 patients with severe combined immunodeficiency: clinical and laboratory features and outcomes. J Clin Immunol 41(7):1563–1573. https://doi.org/10.1007/s10875-021-01062-y

Article  CAS  PubMed  Google Scholar 

Lankester AC, Neven B, Mahlaoui N, von Asmuth ECJ, Courteille V, Alligon M (2022) Hematopoietic cell transplantation in severe combined immunodeficiency: The SCETIDE 2006–2014 European cohort. J Allergy Clin Immunol 149(5):1744–1754.e8. https://doi.org/10.1016/j.jaci.2021.10.017. Epub: 20211027; PubMed PMID: 34718043

Article  CAS  PubMed  Google Scholar 

Thakar MS, Logan BR, Puck JM, Dunn EA, Buckley RH, Cowan MJ et al (2023) Measuring the effect of newborn screening on survival after haematopoietic cell transplantation for severe combined immunodeficiency: a 36-year longitudinal study from the Primary Immune Deficiency Treatment Consortium. Lancet 402(10396):129–140. https://doi.org/10.1016/S0140-6736(23)00731-6. Epub: 20230620; PubMed PMID: 37352885; PubMed Central PMCID: PMC10386791

Article  CAS  PubMed  Google Scholar 

Ikinciogullari A, Cagdas D, Dogu F, Tugrul T, Karasu G, Haskologlu S et al (2019) Clinical features and HSCT outcome for SCID in Turkey. J Clin Immunol. https://doi.org/10.1007/s10875-019-00610-x

Article  PubMed  Google Scholar 

Myers LA, Patel DD, Puck JM, Buckley RH (2002) Hematopoietic stem cell transplantation for severe combined immunodeficiency in the neonatal period leads to superior thymic output and improved survival. Blood 99(3):872–878. https://doi.org/10.1182/blood.V99.3.872

Article  CAS  PubMed  Google Scholar 

Miyamoto S, Umeda K, Kurata M, Nishimura A, Yanagimachi M, Ishimura M et al (2021) Hematopoietic cell transplantation for severe combined immunodeficiency patients: a Japanese retrospective study. J Clin Immunol 41(8):1865–1877. https://doi.org/10.1007/s10875-021-01112-5

Article  CAS  PubMed  PubMed Central  Google Scholar 

Hassan A, Lee P, Maggina P, Xu JH, Moreira D, Slatter M (2014) Host natural killer immunity is a key indicator of permissiveness for donor cell engraftment in patients with severe combined immunodeficiency. J Allergy Clin Immunol 133(6):1660–1666. https://doi.org/10.1016/j.jaci.2014.02.042. Epub: 20140501; PubMed PMID: 24794685; PubMed Central PMCID: PMC4048544

Article  CAS  PubMed  PubMed Central  Google Scholar 

Railey MD, Lokhnygina Y, Buckley RH (2009) Long-term clinical outcome of patients with severe combined immunodeficiency who received related donor bone marrow transplants without pretransplant chemotherapy or post-transplant GVHD prophylaxis. J Pediatr 155(6):834-840.e1. https://doi.org/10.1016/j.jpeds.2009.07.049. Epub: 20091009; PubMed PMID: 19818451; PubMed Central PMCID: PMC2784223

Article  PubMed  PubMed Central  Google Scholar 

Antoine C, Müller S, Cant A, Cavazzana-Calvo M, Veys P, Vossen J et al (2003) Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968–99. Lancet 361(9357):553–560. https://doi.org/10.1016/s0140-6736(03)12513-5. PubMed PMID: 12598139

Article  PubMed  Google Scholar 

Fernandes JF, Rocha V, Labopin M, Neven B, Moshous D, Gennery AR (2012) Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood? Blood 119(12):2949–2955. https://doi.org/10.1182/blood-2011-06-363572. Epub: 20120203; PubMed PMID: 22308292

Article  CAS  PubMed  Google Scholar 

Buckley RH (2011) Transplantation of hematopoietic stem cells in human severe combined immunodeficiency: long term outcomes. Immunol Res 49(1–3):25–43. https://doi.org/10.1007/s12026-010-8191-9.PubMedPMID:21116871;PubMedCentralPMCID:PMC3798033

Article  PubMed  PubMed Central  Google Scholar 

Pai SY, Logan BR, Griffith LM, Buckley RH, Parrott RE, Dvorak CC et al (2014) Transplantation outcomes for severe combined immunodeficiency, 2000–2009. N Engl J Med 371(5):434–446. https://doi.org/10.1056/NEJMoa1401177.PubMedPMID:25075835;PubMedCentralPMCID:PMC4183064

Article  CAS  PubMed  PubMed Central  Google Scholar 

Mazzolari E, De Martiis D, Forino C, Lanfranchi A, Giliani S, Marzollo R et al (2009) Single-center analysis of long-term outcome after hematopoietic cell transplantation in children with congenital severe T cell immunodeficiency. Immunol Res 44(1–3):4–17. https://doi.org/10.1007/s12026-008-8022-4

Article  CAS  PubMed  Google Scholar 

Gennery AR, Slatter MA, Grandin L, Taupin P, Cant AJ, Veys P et al (2010) Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: entering a new century, do we do better? Allergy Clin Immunol 126(3):602–610. https://doi.org/10.1016/j.jaci.2010.06.015. Epub: 20100731; PubMed PMID: 20673987

Article  Google Scholar 

Yao CM, Han XH, Zhang YD, Zhang H, Jin YY, Cao RM et al (2013) Clinical characteristics and genetic profiles of 44 patients with severe combined immunodeficiency (SCID): Report from Shanghai, China (2004–2011). J Clin Immunol 33(3):526–539. https://doi.org/10.1007/s10875-012-9854-1

Article  CAS  PubMed  Google Scholar 

Vignesh P, Rawat A, Kumrah R, Singh A, Gummadi A, Sharma M et al (2021) Clinical, immunological, and molecular features of severe combined immune deficiency: a multi-institutional experience from India. Front immunol 11. https://doi.org/10.3389/fimmu.2020.619146. PubMed PMID: WOS:000620034200001

Aluri J, Desai M, Gupta M, Dalvi A, Terance A, Rosenzweig SD et al (2019) Clinical, immunological, and molecular findings in 57 patients with severe combined immunodeficiency (SCID) from India. Front immunol. https://doi.org/10.3389/fimmu.2019.00023

Fazlollahi MR, Pourpak Z, Hamidieh AA, Movahedi M, Houshmand M, Badalzadeh M et al (2017) Clinical, laboratory, and molecular findings for 63 patients with severe combined immunodeficiency: a decade’s experience. J Investig Allergol Clin Immunol 27(5):299–304. https://doi.org/10.18176/jiaci.0147

Article  CAS  PubMed  Google Scholar 

留言 (0)

沒有登入
gif