Pre-Transplant Immune Dysregulation Predicts for Poor Outcome Following Allogeneic Haematopoietic Stem Cell Transplantation in Adolescents and Adults with Inborn Errors of Immunity (IEI)

Bousfiha A, Moundir A, Tangye SG, Picard C, Jeddane L, Al-Herz W, et al. The 2022 update of IUIS Phenotypical classification for human inborn errors of immunity. J Clin Immunol. 2022;42(7):1508–20.

Article  PubMed  Google Scholar 

Bousfiha A, Jeddane L, Picard C, Al-Herz W, Ailal F, Chatila T, et al. Human inborn errors of immunity: 2019 update of the IUIS Phenotypical classification. J Clin Immunol. 2020;40(1):66–81.

Article  PubMed  PubMed Central  Google Scholar 

Fox TA, Chakraverty R, Burns S, Carpenter B, Thomson K, Lowe D, et al. Successful outcome following allogeneic hematopoietic stem cell transplantation in adults with primary immunodeficiency. Blood. 2018;131(8):917–31.

Article  PubMed  PubMed Central  CAS  Google Scholar 

Liese J, Kloos S, Jendrossek V, Petropoulou T, Wintergerst U, Notheis G, et al. Long-term follow-up and outcome of 39 patients with chronic granulomatous disease. J Pediatr. 2000;137(5):687–93.

Article  PubMed  CAS  Google Scholar 

Gatti RA, Meuwissen HJ, Allen HD, Hong R, Good RA. Immunological reconstitution of sex-linked lymphopenic immunological deficiency. Lancet. 1968;2(7583):1366–9.

Article  PubMed  CAS  Google Scholar 

Bach FH, Albertini RJ, Joo P, Anderson JL, Bortin MM. Bone-marrow transplantation in a patient with the Wiskott-Aldrich syndrome. Lancet. 1968;2(7583):1364–6.

Article  PubMed  CAS  Google Scholar 

Fischer A, Landais P, Friedrich W, Morgan G, Gerritsen B, Fasth A, et al. European experience of bone-marrow transplantation for severe combined immunodeficiency. Lancet. 1990;336(8719):850–4.

Article  PubMed  CAS  Google Scholar 

Fischer A, Landais P, Friedrich W, Gerritsen B, Fasth A, Porta F, et al. Bone marrow transplantation (BMT) in Europe for primary immunodeficiencies other than severe combined immunodeficiency: a report from the European Group for BMT and the European Group for Immunodeficiency. Blood. 1994;83(4):1149–54.

Article  PubMed  CAS  Google Scholar 

Pai SY, Cowan MJ. Stem cell transplantation for primary immunodeficiency diseases: the north American experience. Curr Opin Allergy Clin Immunol. 2014;14(6):521–6.

Article  PubMed  PubMed Central  CAS  Google Scholar 

Burroughs LM, Petrovic A, Brazauskas R, Liu X, Griffith LM, Ochs HD, et al. Excellent outcomes following hematopoietic cell transplantation for Wiskott-Aldrich syndrome: a PIDTC report. Blood. 2020;135(23):2094–105.

Article  PubMed  PubMed Central  Google Scholar 

Moratto D, Giliani S, Bonfim C, Mazzolari E, Fischer A, Ochs HD, et al. Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980–2009: an international collaborative study. Blood. 2011;118(6):1675–84.

Article  PubMed  PubMed Central  CAS  Google Scholar 

Ozsahin H, Cavazzana-Calvo M, Notarangelo LD, Schulz A, Thrasher AJ, Mazzolari E, et al. Long-term outcome following hematopoietic stem-cell transplantation in Wiskott-Aldrich syndrome: collaborative study of the European Society for Immunodeficiencies and European Group for blood and marrow transplantation. Blood. 2008;111(1):439–45.

Article  PubMed  CAS  Google Scholar 

Ngwube A, Hanson IC, Orange J, Rider NL, Seeborg F, Shearer W, et al. Outcomes after allogeneic transplant in patients with Wiskott-Aldrich Syndrome. Biol Blood Marrow Transpl. 2018;24(3):537–41.

Article  Google Scholar 

Gungor T, Teira P, Slatter M, Stussi G, Stepensky P, Moshous D, et al. Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study. Lancet. 2014;383(9915):436–48.

Article  PubMed  CAS  Google Scholar 

Chiesa R, Wang J, Blok HJ, Hazelaar S, Neven B, Moshous D, et al. Hematopoietic cell transplantation in chronic granulomatous disease: a study of 712 children and adults. Blood. 2020;136(10):1201–11.

Article  PubMed  Google Scholar 

Soncini E, Slatter MA, Jones LB, Hughes S, Hodges S, Flood TJ, et al. Unrelated donor and HLA-identical sibling haematopoietic stem cell transplantation cure chronic granulomatous disease with good long-term outcome and growth. Br J Haematol. 2009;145(1):73–83.

Article  PubMed  CAS  Google Scholar 

Burns SO, Morris EC. How I use allogeneic HSCT for adults with inborn errors of immunity. Blood. 2021;138(18):1666–76.

Article  PubMed  CAS  Google Scholar 

Pai SY, Logan BR, Griffith LM, Buckley RH, Parrott RE, Dvorak CC, et al. Transplantation outcomes for severe combined immunodeficiency, 2000–2009. N Engl J Med. 2014;371(5):434–46.

Article  PubMed  PubMed Central  CAS  Google Scholar 

Bortoletto P, Lyman K, Camacho A, Fricchione M, Khanolkar A, Katz BZ. Chronic granulomatous disease: a large, single-center US experience. Pediatr Infect Dis J. 2015;34(10):1110–4.

Article  PubMed  PubMed Central  Google Scholar 

Morris EC, Albert MH. Allogeneic HSCT in adolescents and young adults with primary immunodeficiencies. Front Pediatr. 2019;7:437.

Article  PubMed  PubMed Central  Google Scholar 

Marcais A, Mahlaoui N, Neven B, Lanternier F, Catherinot E, Salvator H, et al. Curative allogeneic hematopoietic stem cell transplantation following reduced toxicity conditioning in adults with primary immunodeficiency. Bone Marrow Transpl. 2022;57(10):1520–30.

Article  CAS  Google Scholar 

Dimitrova D, Gea-Banacloche J, Steinberg SM, Sadler JL, Hicks SN, Carroll E, et al. Prospective study of a Novel, Radiation-Free, reduced-intensity bone marrow transplantation platform for primary Immunodeficiency diseases. Biol Blood Marrow Transpl. 2020;26(1):94–106.

Article  CAS  Google Scholar 

Albert MH, Hauck F, Wiebking V, Aydin S, Notheis G, Koletzko S, et al. Allogeneic stem cell transplantation in adolescents and young adults with primary immunodeficiencies. J Allergy Clin Immunol Pract. 2018;6(1):298–e3012.

Article  PubMed  Google Scholar 

Albert MH, Sirait T, Eikema DJ, Bakunina K, Wehr C, Suarez F, et al. Hematopoietic stem cell transplantation for adolescents and adults with inborn errors of immunity: an EBMT IEWP study. Blood. 2022;140(14):1635–49.

Article  PubMed  CAS  Google Scholar 

Cheminant M, Fox TA, Alligon M, Bouaziz O, Neven B, Moshous D, et al. Allogeneic stem cell transplantation compared to conservative management in adults with inborn errors of immunity. Blood. 2023;141(1):60–71.

Article  PubMed  CAS  Google Scholar 

Thakar MS, Broglie L, Logan B, Artz A, Bunin N, Burroughs LM, et al. The hematopoietic cell transplant Comorbidity Index predicts survival after allogeneic transplant for nonmalignant diseases. Blood. 2019;133(7):754–62.

Article  PubMed  PubMed Central  CAS  Google Scholar 

Seidel MG, Tesch VK, Yang LN, Hauck F, Horn AL, Smolle MA, et al. The Immune Deficiency and Dysregulation Activity (IDDA2.1 ‘Kaleidoscope’) score and other clinical measures in inborn errors of immunity (Nov, 10.1007/s10875-021-01177-2, 2021). J Clin Immunol. 2022;42(3):499.

Article  PubMed  PubMed Central  Google Scholar 

Tesch VK, Abolhassani H, Shadur B, Zobel J, Mareika Y, Sharapova S, et al. Long-term outcome of LRBA deficiency in 76 patients after various treatment modalities as evaluated by the immune deficiency and dysregulation activity (IDDA) score. J Allergy Clin Immun. 2020;145(5):1452–63.

Article  PubMed  CAS  Google Scholar 

Tazoe K, Harada N, Makuuchi Y, Kuno M, Takakuwa T, Okamura H, et al. Systemic inflammatory autoimmune disease before allogeneic hematopoietic stem cell transplantation is a risk factor for death in patients with myelodysplastic syndrome or chronic myelomonocytic leukemia. Ann Hematol. 2024;103(6):2059–72.

Article  PubMed  CAS  Google Scholar 

Kong Y, Wang YT, Cao XN, Song Y, Chen YH, Sun YQ, et al. Aberrant T cell responses in the bone marrow microenvironment of patients with poor graft function after allogeneic hematopoietic stem cell transplantation. J Transl Med. 2017;15(1):57.

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