Hematopoietic cell transplantation in severe combined immunodeficiency: the SCETIDE 2006-2014 European cohort

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Abstract: Background
Hematopoietic stem cell transplantation (HSCT) represents a curative treatment for patients with severe combined immunodeficiency (SCID), a group of monogenic immune disorders with an otherwise fatal outcome.

Objective
To perform a comprehensive multicenter analysis of genotype-specific HSCT outcome including detailed analysis of immune reconstitution (IR) and the predictive value for clinical outcome.

Methods
HSCT outcome was studied in 338 patients with genetically confirmed SCID, transplanted in 2006-2014 and registered in the SCETIDE registry. In a representative subgroup of n=152 patients data on IR and long-term clinical outcome were analyzed.

Results
2-years OS was similar with matched family and unrelated donors and superior to mismatched donor HSCT (p < 0.001). The 2-year EFS was similar in matched and mismatched unrelated donor and less favorable in mismatched related donor (MMRD) HSCT (p < 0.001). Genetic subgroups did not differ in 2-year OS (p=0.1) and EFS (p=0.073). In multivariate analysis, pretransplant infections and use of MMRD were associated with less favorable OS and EFS. With a median follow-up of 6.2 years [range 2.0-11.8 years], 73/152 IR cohort patients were alive and well without immunoglobulin dependency. IL2Rγ-JAK3-IL7R deficient SCID, myeloablative conditioning, matched donor HSCT, and naïve CD4 T lymphocytes > 0.5x10e3/μL at +1-year were identified as independent predictors of favorable clinical and immunological outcome.

Conclusion
Recent advances in HSCT in SCID patients have resulted in improved OS and EFS in all genotypes and donor types. To achieve a favorable long term outcome, treatment strategies should aim for optimal naïve CD4 T lymphocyte regeneration

Location
Deutsche Nationalbibliothek Frankfurt am Main
Extent
Online-Ressource
Language
Englisch
Notes
The journal of allergy and clinical immunology. - 149, 5 (2022) , 1744-1754.e8, ISSN: 1097-6825

Event
Veröffentlichung
(where)
Freiburg
(who)
Universität
(when)
2021
Creator
Lankester, Arjan C.
Neven, Benedicte
Speckmann, Carsten
Fischer, Alain
Gennery, Andrew R.

DOI
10.1016/j.jaci.2021.10.017
URN
urn:nbn:de:bsz:25-freidok-2226325
Rights
Open Access; Der Zugriff auf das Objekt ist unbeschränkt möglich.
Last update
25.03.2025, 1:51 PM CET

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Associated

  • Lankester, Arjan C.
  • Neven, Benedicte
  • Speckmann, Carsten
  • Fischer, Alain
  • Gennery, Andrew R.
  • Universität

Time of origin

  • 2021

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