Hematopoietic stem cell transplantation in systemic autoinflammatory diseases - the first one hundred transplanted patients.
Expert Rev Clin Immunol
; 18(7): 667-689, 2022 07.
Article
em En
| MEDLINE
| ID: mdl-35575194
ABSTRACT
INTRODUCTION:
Primary immune regulatory disorders encompass a range of clinical conditions caused by different defects of immune regulatory mechanisms, including systemic autoinflammatory diseases (AIDs). Allogeneic hematopoietic stem cell transplantation may be a therapeutic option for AIDs, particularly if response to conventional treatments is lacking. AREAS COVERED HSCT has been reported as a possible therapeutic option in several AID subgroups, namely, inflammasomopathies, immuno-proteinopathies,actinopathies, relopathies, interferonopathies, and Adenosine Deaminase 2 deficiency. Here, an extensive review of the literature summarizes the available data on HSCT outcome in AIDs. EXPERT OPINION HSCT in AIDs is mainly indicated in case of ineffectiveness of conventional therapies and/or co-existence of immunodeficiency in conditions characterized by a primary involvement of the hematopoietic compartment. An effective control of the inflammatory process before HSCT reduces the risk of alloreactivity. HLA identical family donor represents the first choice, but in most cases it is essential to exclude a possible carrier status. If a suitable HLA identical family donor is not available, a haploidentical donor with platform with T-depletion could offer some benefit lowering the risk of GvHD. Treosulfan-based conditioning regimens could be recommended to reduce toxicity and prevent rejection. Target chimerism may differ based on the primary disease's pathogenic mechanism.Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Contexto em Saúde:
2_ODS3
Base de dados:
MEDLINE
Assunto principal:
Síndrome da Imunodeficiência Adquirida
/
Transplante de Células-Tronco Hematopoéticas
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Doenças Hereditárias Autoinflamatórias
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Doença Enxerto-Hospedeiro
Limite:
Humans
Idioma:
En
Revista:
Expert Rev Clin Immunol
Ano de publicação:
2022
Tipo de documento:
Article