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Non-osteopenic Bone Pathology After Allo-hematopoietic Stem Cell Transplantation in Patients with Inborn Errors of Immunity.
Golwala, Zainab M; Bhat, Nikita Gireesh; Xu-Bayford, Jinhua; Stankova, Tanja; Adams, Stuart; Morris, Emma C; Qasim, Waseem; Booth, Claire; Worth, Austen; Kusters, Maaike A; Elfeky, Reem.
Afiliação
  • Golwala ZM; Department of Immunology, Great Ormond Street Hospital for Children NHS Foundation Trust, Great Ormond Street, London, WC1N 3JH, UK.
  • Bhat NG; UCL Great Ormond Street Institute of Child Health, London, UK.
  • Xu-Bayford J; Department of Immunology, Great Ormond Street Hospital for Children NHS Foundation Trust, Great Ormond Street, London, WC1N 3JH, UK.
  • Stankova T; Department of Immunology, Great Ormond Street Hospital for Children NHS Foundation Trust, Great Ormond Street, London, WC1N 3JH, UK.
  • Adams S; Department of Immunology, Great Ormond Street Hospital for Children NHS Foundation Trust, Great Ormond Street, London, WC1N 3JH, UK.
  • Morris EC; SIHMDS-Haematology, Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK.
  • Qasim W; Department of Immunology, Institute of Immunity and Transplantation, University College London, London, UK.
  • Booth C; Department of Immunology, Great Ormond Street Hospital for Children NHS Foundation Trust, Great Ormond Street, London, WC1N 3JH, UK.
  • Worth A; UCL Great Ormond Street Institute of Child Health, London, UK.
  • Kusters MA; Department of Immunology, Great Ormond Street Hospital for Children NHS Foundation Trust, Great Ormond Street, London, WC1N 3JH, UK.
  • Elfeky R; UCL Great Ormond Street Institute of Child Health, London, UK.
J Clin Immunol ; 43(5): 1019-1031, 2023 07.
Article em En | MEDLINE | ID: mdl-36930409
ABSTRACT

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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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Síndrome de Wiskott-Aldrich / Transplante de Células-Tronco Hematopoéticas / Linfo-Histiocitose Hemofagocítica Tipo de estudo: Etiology_studies / Incidence_studies / Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Child / Humans Idioma: En Ano de publicação: 2023 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Síndrome de Wiskott-Aldrich / Transplante de Células-Tronco Hematopoéticas / Linfo-Histiocitose Hemofagocítica Tipo de estudo: Etiology_studies / Incidence_studies / Observational_studies / Prognostic_studies / Risk_factors_studies Limite: Child / Humans Idioma: En Ano de publicação: 2023 Tipo de documento: Article