Your browser doesn't support javascript.
loading
Autologous haematopoiesis stem cell transplantation (AHSCT) for treatment-refractory autoimmune diseases in children.
Satirer, Özlem; Henes, Joerg C; Döring, Michaela; Lesk, Till; Benseler, Susanne; Kuemmerle-Deschner, Jasmin Beate.
Afiliación
  • Satirer Ö; Department of Paediatrics and Autoinflammation reference Center Tuebingen (arcT), Universitatsklinikum Tubingen, Tubingen, Baden-Württemberg, Germany.
  • Henes JC; Centre for Interdisciplinary Clinical Immunology, Rheumatology and Auto-inflammatory Diseases and Department of Internal Medicine II (Oncology, Haematology, Immunology and Rheumatology), Universitatsklinikum Tubingen, Tubingen, Baden-Württemberg, Germany.
  • Döring M; Pediatric Hematology &Oncology, University of Tübingen, Tubingen, Baden-Württemberg, Germany.
  • Lesk T; Universitatsklinikum Tubingen, Tubingen, Baden-Württemberg, Germany.
  • Benseler S; Rheumatology, Alberta Children's Hospital Research Institute, Calgary, Alberta, Canada.
  • Kuemmerle-Deschner JB; Children's Health Ireland, Dublin, Dublin, Ireland.
RMD Open ; 10(3)2024 Jul 14.
Article en En | MEDLINE | ID: mdl-39004431
ABSTRACT

OBJECTIVES:

To evaluate the long-term effectiveness and safety of autologous haematopoiesis stem cell transplantation (AHSCT) for severe, refractory autoimmune diseases in paediatric patients.

METHODS:

A single-centre study of consecutive children and adolescents with refractory autoimmune diseases undergoing AHSCT was performed. Demographics, clinical, laboratory features, pre-AHSCT medications, disease activity and functional status were captured. The primary outcome was progression-free survival, secondary outcomes included overall survival, disease-specific treatment responses, disease activity at the last follow-up and AHSCT safety.

RESULTS:

The study included seven patients two systemic sclerosis, one pansclerotic morphoea, one eosinophilic fasciitis, one juvenile dermatomyositis and two patients with systemic juvenile idiopathic arthritis; four women, three men median age at AHSCT of 10 years (7-19), median follow-up post-AHSCT of 17 years. Median progression-free survival and overall survival was 4.2 years (95% CI 0.98 to 8.3) and 17 years (95% CI 11.8 to 22.1), respectively. Progression-free survival rates at 1 and 2 years post-AHSCT were 100% and 77%, respectively. All children survived. All patients are in clinical remission, only four require ongoing immunotherapy. SAFETY Three experienced infections, including HHV6, Candida and Ralstonia sepsis; one developed a systemic inflammatory response syndrome; two new onset secondary autoimmune diseases including autoimmune haemolytic anaemia, Graves' disease and one was found to have a breast fibroadenoma. Treatment toxicity one cyclophosphamide-associated transient renal failure and pericardial effusion, one patient with amenorrhoea/infertility.

CONCLUSIONS:

AHSCT was an effective and safe approach for children and adolescents with treatment-refractory autoimmune diseases. The indication and timing of transplantation requires a careful consideration and a multidisciplinary approach.
Asunto(s)
Palabras clave

Texto completo: 1 Base de datos: MEDLINE Asunto principal: Enfermedades Autoinmunes / Trasplante Autólogo / Trasplante de Células Madre Hematopoyéticas Límite: Adolescent / Adult / Child / Female / Humans / Male Idioma: En Revista: RMD Open Año: 2024 Tipo del documento: Article País de afiliación: Alemania

Texto completo: 1 Base de datos: MEDLINE Asunto principal: Enfermedades Autoinmunes / Trasplante Autólogo / Trasplante de Células Madre Hematopoyéticas Límite: Adolescent / Adult / Child / Female / Humans / Male Idioma: En Revista: RMD Open Año: 2024 Tipo del documento: Article País de afiliación: Alemania