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Non-osteopenic Bone Pathology After Allo-hematopoietic Stem Cell Transplantation in Patients with Inborn Errors of
Zainab M Golwala1,2, Nikita Gireesh Bhat1, Jinhua Xu-Bayford1
1Department of Immunology, Great Ormond Street Hospital for Children NHS Foundation Trust, Great Ormond Street, London, WC1N 3JH, UK.
Insights
Non-osteopenic bone pathology is a significant concern for children surviving hematopoietic stem cell transplant (HSCT) for inborn errors of immunity (IEI). Regular bone health assessments are crucial, especially for Wiskott-Aldrich syndrome patients.
Area of Science:
- Pediatric Immunology
- Hematology
- Orthopedics
- Genetics
Background:
- Hematopoietic stem cell transplantation (HSCT) is a life-saving treatment for children with inborn errors of immunity (IEI).
- Long-term complications of HSCT, particularly non-osteopenic bone pathology, are not well-characterized in pediatric IEI populations.
- Understanding post-HSCT bone complications is vital for improving long-term outcomes and patient care.
Purpose of the Study:
- To investigate the incidence and characteristics of non-osteopenic bone pathology in children with IEI following allogeneic HSCT (allo-HSCT).
- To identify potential risk factors and specific patient groups at higher risk for developing bone pathologies post-HSCT.
- To emphasize the need for ongoing bone health surveillance in long-term survivors of HSCT for IEI.
Main Methods:
- A descriptive study analyzing data from children with IEI who underwent allo-HSCT between 2000 and 2018.
- Bone pathologies were categorized into bone tumors, skeletal dysplasia, avascular necrosis, evolving bone deformities, and slipped upper femoral epiphysis.
- Exclusion criteria focused on isolating non-osteopenic pathologies from conditions like isolated reduced bone density or IEI-related skeletal issues.
Main Results:
- Non-osteopenic bone pathology was observed in 9.4% of long-term survivors (32/340), with a mean onset of 7.8 years post-HSCT.
- The majority of affected patients (81.2%) received treosulfan-based conditioning, and 65.6% had a history of prolonged steroid use.
- Wiskott-Aldrich syndrome (WAS) showed the highest incidence (23.5%), followed by hemophagocytic lymphohistiocytosis (18.8%). Pain was the primary symptom in 66% of cases.
Conclusions:
- Non-osteopenic bone pathology is a significant, albeit not rare, complication in long-term survivors of allo-HSCT for IEI.
- The findings underscore the necessity for continuous bone health monitoring, especially in patients with IEI, given the delayed presentation of symptoms.
- Children with stunted growth post-HSCT require skeletal surveys to detect potential skeletal dysplasia, with WAS patients needing particular attention.
Purpose:
There is a lack of data on post-HSCT non-osteopenic bone pathology specifically for children with inborn errors of immunity (IEI). We collected data on non-osteopenic bone pathology in children with IEI post-HSCT over two decades in a large tertiary pediatric immunology center.
Methods:
Descriptive study with data analysis of bone pathology in allo-HSCT for IEI was performed between 1/1/2000 to 31/12/2018 including patients alive at follow-up to July 2022. Records were analyzed for bone pathology and risk factors. Exclusion criteria included isolated reduced bone density, fractures, and skeletal anomalies due to underlying IEI and short stature without other bone pathology. Bone pathologies were divided into 5 categories: bone tumors; skeletal dysplasia; avascular necrosis; evolving bone deformities; slipped upper femoral epiphysis.
Results:
A total of 429 children received HSCT between 2000 and 2018; 340 are alive at last assessment. Non-osteopenic bone pathology was observed post-HSCT in 9.4% of patients (32/340, mean 7.8 years post-HSCT). Eleven patients (34%) had > 1 category of bone pathology. Seventeen patients (17/32; 53%) presented with bilateral bone pathology. The majority of patients received treosulfan-based conditioning (26/32; 81.2%). Totally, 65.6% (21/32) of patients had a history of prolonged steroid use (> 6 months). Pain was the presenting symptom in 66% of patients, and surgical intervention was required in 43.7%. The highest incidence of bone pathologies was seen in Wiskott-Aldrich syndrome (WAS) (n = 8/34; 23.5%) followed by hemophagocytic lymphohistiocytosis patients (n = 3/16; 18.8%).
Conclusion:
Non-osteopenic bone pathology in long-term survivors of allo-HSCT for IEI is not rare. Most patients did not present with complaints until at least 5 years post-HSCT highlighting the need for ongoing bone health assessment for patients with IEI. Children presenting with stunted growth and bone pathology post-HSCT should undergo skeletal survey to rule out development of post-HSCT skeletal dysplasia. Increased rates and complexity of bone pathology were seen amongst patients with Wiskott-Aldrich syndrome.
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