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Posttransplantation late complications increase over time for patients with SCID: A Primary Immune Deficiency
Hesham Eissa1, Monica S Thakar2, Ami J Shah3
1Division of Pediatric Hematology-Oncology-BMT, University of Colorado, Aurora, Wash.
Insights
Hematopoietic cell transplantation (HCT) for severe combined immunodeficiency (SCID) leads to substantial chronic and late effects (CLE), impacting survival. Developing survivorship guidelines is crucial for managing long-term outcomes in SCID HCT survivors.
Area of Science:
- Immunology
- Pediatric Hematology
- Transplantation Medicine
Background:
- The Primary Immune Deficiency Treatment Consortium (PIDTC) conducted a natural history study on hematopoietic cell transplantation (HCT) for children in the US and Canada.
- Severe combined immunodeficiency (SCID) is a critical condition requiring HCT for survival.
Purpose of the Study:
- To investigate the outcomes of HCT for SCID.
- To evaluate the chronic and late effects (CLE) following HCT in pediatric SCID patients.
Main Methods:
- Retrospective analysis of 399 SCID patients who underwent HCT between 1982 and 2012 at 32 PIDTC centers.
- Defined CLE as conditions present before 2 years post-HCT (chronic) or developing after 2 years (late).
- Eligibility required survival to at least 2 years post-HCT without further cellular therapy.
Main Results:
- Cumulative incidence of CLE reached 41% by 15 years post-HCT, with neurologic, neurodevelopmental, and dental issues being most common.
- Chemotherapy conditioning was linked to reduced height and increased endocrine/dental CLE.
- Presence of any CLE significantly increased the risk of late death (HR 7.21).
Conclusions:
- Late morbidity after HCT for SCID is significant and adversely affects overall survival.
- Evidence supports the development of survivorship guidelines tailored to SCID HCT recipients.
- Long-term monitoring and management are essential for improving outcomes in these patients.
Background:
The Primary Immune Deficiency Treatment Consortium (PIDTC) enrolled children in the United States and Canada onto a retrospective multicenter natural history study of hematopoietic cell transplantation (HCT).
Objective:
We investigated outcomes of HCT for severe combined immunodeficiency (SCID).
Methods:
We evaluated the chronic and late effects (CLE) after HCT for SCID in 399 patients transplanted from 1982 to 2012 at 32 PIDTC centers. Eligibility criteria included survival to at least 2 years after HCT without need for subsequent cellular therapy. CLE were defined as either conditions present at any time before 2 years from HCT that remained unresolved (chronic), or new conditions that developed beyond 2 years after HCT (late).
Results:
The cumulative incidence of CLE was 25% in those alive at 2 years, increasing to 41% at 15 years after HCT. CLE were most prevalent in the neurologic (9%), neurodevelopmental (8%), and dental (8%) categories. Chemotherapy-based conditioning was associated with decreased-height z score at 2 to 5 years after HCT (P < .001), and with endocrine (P < .001) and dental (P = .05) CLE. CD4 count of ≤500 cells/microL and/or continued need for immunoglobulin replacement therapy >2 years after transplantation were associated with lower-height z scores. Continued survival from 2 to 15 years after HCT was 90%. The presence of any CLE was associated with increased risk of late death (hazard ratio, 7.21; 95% confidence interval, 2.71-19.18; P < .001).
Conclusion:
Late morbidity after HCT for SCID was substantial, with an adverse impact on overall survival. This study provides evidence for development of survivorship guidelines based on disease characteristics and treatment exposure for patients after HCT for SCID.
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