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Hematopoietic stem cell transplantation for Crohn's disease: Gaps, doubts and perspectives
Milton Artur Ruiz1, Roberto Luiz Kaiser Junior2, Lilian Piron-Ruiz2
1Bone Marrow Transplant Department, Associação Portuguesa de Beneficência, São José do Rio Preto, SP 15090 470, Brazil. milruiz@yahoo.com.br.
Insights
Cell therapy, including hematopoietic stem cell transplantation, offers a new treatment option for Crohn's disease (CD). While promising, further research is needed to address knowledge gaps and refine its application for this inflammatory bowel disease.
Area of Science:
- Gastroenterology and Immunology
- Regenerative Medicine
Background:
- Crohn's disease (CD) is a chronic inflammatory bowel disease characterized by immunological imbalance, leading to intestinal lesions and complications.
- Current treatments aim to manage symptoms and heal lesions, often involving surgical procedures.
- Cell therapy, initially explored in cancer patients, has emerged as a potential therapeutic avenue for CD.
Discussion:
- The use of cell therapy for CD evolved from treating co-occurring conditions like lymphoma to directly addressing the disease.
- Hematopoietic stem cell transplantation (HSCT) in its autologous form is the primary cell therapy modality currently used for CD.
- Mesenchymal stromal cells are being experimentally investigated for treating specific CD-related lesions, particularly in the anal mucosa.
Key Insights:
- Cell therapy, particularly autologous HSCT, has shown promise in improving CD symptoms and healing intestinal lesions.
- The application of cell therapy for CD has expanded since 2003, with published case series and randomized studies.
- Despite positive outcomes, significant knowledge gaps and uncertainties persist regarding the optimal use and efficacy of cell therapy in CD management.
Outlook:
- Future research should focus on elucidating the precise mechanisms of action for cell therapy in CD.
- Standardizing mobilization regimens and conditioning protocols for HSCT in CD patients is crucial.
- Further clinical trials are needed to establish cell therapy as a definitive treatment and explore novel cell types for CD management.
Abstract:
Crohn's disease (CD) is an inflammatory bowel disease that can affect any site of the digestive system. It occurs due to an immunological imbalance and is responsible for intestinal mucosal lesions and complications such as fistulas and stenoses. Treatment aims to stabilize the disease, reducing the symptoms and healing intestinal lesions. Surgical procedures are common in patients. Cell therapy was initially used to treat this disease in patients who also suffered from lymphoma and leukemia and were considered to be good candidates for autologous and allogeneic transplantation. After transplantation, an improvement was also observed in their CD. In 2003, the procedure began to be used to treat the disease itself, and several case series and randomized studies have been published since then; this approach currently comprises a new option in the treatment of CD. However, considerable doubt along with significant gaps in our knowledge continue to exist in relation to cell therapy for CD. Cell therapy is currently restricted to the autologous modality of hematopoietic stem cell transplantation and, experimentally, to mesenchymal stromal cells to directly treat lesions of the anal mucosa. This article presents the supporting claims for transplantation as well as aspects related to the mobilization regime, conditioning and perspectives of cell therapy.