Adult patients with inborn errors of immunity (IEI) who underwent an allogeneic stem cell transplant (alloSCT) were less likely to develop progressive morbidity compared with adults treated with conservative measures, according to the results of a retrospective study published Blood.
Although alloSCT is considered curative for IEI, “questions remain regarding the indications for and optimal timing of transplant,” the authors wrote in their report. This retrospective study evaluated data from 79 adult patients with severe IEI who underwent an alloSCT between 2008 and 2018 and a matched cohort of 202 nontransplanted adults with severe IEI. The median follow-up of 4.8 years.
The median age of both cohorts was 25. In the transplanted group, the most common reasons for referral were lymphoproliferative disease in 28% and gastrointestinal complications in 19%. Additional indications included infection, invasive aspergillosis, autoimmune neutropenia, and liver involvement, among others.
The 1-year transplant-related mortality was 13% and the projected 5-year cumulative mortality rate was 30% in the alloSCT group compared with 11% in the nontransplanted group (P <.001). However, overall survival was similar between the groups, with 84% and 90% alive at 1 year in the transplanted and nontransplanted cohorts, respectively.
Mortality within the first year after transplant was significantly associated with invasive aspergillosis (hazard ratio [HR], 8.56; 95% CI, 4.63-15.81; P <.01), autoimmunity (HR, 2.14; 95% CI, 1.31-3.5; P <.01), and lymphoid malignancy (HR, 4.66; 95% CI, 2.99-7.27; P <.01).
Transplant resulted in a significantly higher 5-year disease-free survival at 58% compared with 33% in the nontransplanted cohort (P =.007). At the end of follow-up, there were 90% of patients in remission and 80% in complete remission without transplant-related complications.
The authors used recurrent events to estimate quality of life. Over time, recurrent events improved among patients who underwent transplant, with significantly fewer recurrent events the first year after transplant compared with nontransplanted patients (HR, 0.25; 95% CI, 0.1-0.6; P <.01).
The authors concluded that “alloSCT prevents progressive morbidity associated with IEI, which may outweigh the negative impact of transplant-related mortality.”
Disclosures: Some of the study authors declared affiliations with biotech, pharmaceutical, or device companies. Please see the original reference for a full list of disclosures.
Cheminant M, Fox TA, Aligon M, et al. Allogeneic stem cell transplantation compared to conservative management in adults with inborn errors of immunity. Blood. 2023;141:60-71. doi: 10.1182/blood.2022015482