التفاصيل البيبلوغرافية
| العنوان: |
Transplantation Outcomes for Children with Severe Combined Immune Deficiency (SCID) Have Improved over Time: A 36-Year Summary Report By the Primary Immune Deficiency Treatment Consortium (PIDTC). |
| المؤلفون: |
Thakar, Monica S.1,2 (AUTHOR), Logan, Brent3 (AUTHOR), Buckley, Rebecca H.4 (AUTHOR), Haddad, Elie5 (AUTHOR), Dvorak, Christopher C.6 (AUTHOR), O'Reilly, Richard J.7 (AUTHOR), Kapoor, Neena8 (AUTHOR), Satter, Lisa Forbes9 (AUTHOR), Martinez, Caridad10 (AUTHOR), Pai, Sung-Yun11 (AUTHOR), Heimall, Jennifer12 (AUTHOR), Jyonouchi, Soma12 (AUTHOR), Sullivan, Kathleen E.12 (AUTHOR), Chandra, Sharat13 (AUTHOR), Smith, Angela R.14 (AUTHOR), Chaudhury, Sonali15 (AUTHOR), Saldana, Blachy Davila16 (AUTHOR), Sunkersett, Gauri17 (AUTHOR), Shyr, David C.18 (AUTHOR), Burroughs, Lauri M.19 (AUTHOR) |
| المصدر: |
Biology of Blood & Marrow Transplantation. 2020 Supplement, Vol. 26, pS18-S19. 2p. |
| مصطلحات موضوعية: |
*IMMUNODEFICIENCY, *ADENOSINE deaminase, *GRAFT versus host disease, *CELL transplantation, *KRUSKAL-Wallis Test, *CORD blood |
| مستخلص: |
With >36 years of data collected, PIDTC prospective (6901) and retrospective (6902) natural history studies provide an unprecedented opportunity to study hematopoietic cell transplantation (HCT) outcomes for SCID over time. Patients in this 6901/6902 analysis met PIDTC diagnostic criteria for SCID and underwent HCT. Categorial variables were analyzed between decades (a) 1982-89 (b) 1990-99 (c) 2000-09 (d) 2010-18 using the chi-square test. Continuous outcomes were compared using the Kruskal-Wallis test. Kaplan-Meier method was used for estimates of overall survival (OS). 896 children with typical (n=742) and atypical (n=154) SCID requiring HCT between 1982-2018 were enrolled. Diagnosis of SCID for reasons other than family history or newborn screening was common (60%) in early cohorts (a-c) dropping to 30% in cohort (d). Distribution of SCID genotypes changed over time (Fig 1), and novel/unknown genotypes also decreased from (a) 53% to (d) 13%, p<0.001. Due to the rise of gene therapy, there were fewer patients with adenosine deaminase deficient SCID receiving HCT in cohort (d) compared to prior decades. Median age at HCT has decreased, from 193 to 112 days of life, p<0.001. While marrow was the most common stem cell source in the 1980's (95%), currently peripheral blood stem cells and cord blood are given to 46% of patients (p<0.001). The increased use of unrelated donors parallels a decrease in mismatched related donors (Fig 2a; p<0.001). In univariate analysis, use of conditioning has increased from 12% in the 1980's to 65% currently (Fig 2b, p<0.001); contributing factors that will be evaluated in upcoming multivariate analyses include genotype, typical vs atypical SCID, and donor type. Day +100 cumulative incidence of grades 3-4 acute graft-versus-host disease (GVHD) has dropped, from 9.6% to 5.9% (p=0.02). OS has markedly improved in the last decade (Fig 3a), with 1- and 5-year OS being 90 and 87%, respectively (p=0.02). In particular, gains have been made in OS when using alternative donors (p=0.02) (Fig 3c,d) while OS for matched sibling HCT remains high (Fig 3b). This longitudinal data set, spanning the implementation of NBS and advancements in diagnostics and supportive care, highlights improved OS after HCT for SCID, including alternative donors. Exploration of decreased toxicity approaches that maintain high OS and engraftment are warranted. [ABSTRACT FROM AUTHOR] |
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