RESUMEN
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.
RESUMEN
BACKGROUND: Hematopoietic stem cell transplantation (HSCT) can be used in the treatment of patients with refractory Crohn's disease (CD) when no alternative treatment is available. However, HSCT increases the risk of infections, in particular during the aplasia of mobilization and conditioning. Moreover, intestinal stomas in CD augment the risk of morbidity in immunocompromised patients and under aplastic conditions. The objective of this report was to describe the results of the first year after HSCT in a CD patient with an intestinal stoma. METHODS: The patient was assessed in respect to disease symptoms and endoscopic findings before the procedure and 30, 90, 180, and 365 days after HSCT. RESULTS: No complications were observed during mobilization and conditioning with sufficient CD34+ cells being harvested in just one apheresis session. Toxicity was restricted to the hematological series. Scores of all the CD indexes and the quality of life of the patient improved. However, two of three endoscopic scores remained unchanged even though improvements were found in the appearance of the lesions. CONCLUSION: HSCT may be an alternative treatment for refractory CD in patients with an intestinal stoma, and a priori, carefully selected patients with stomas should not be excluded as candidates for this procedure.
RESUMEN
OBJECTIVE: The incidence of adverse events in myeloablative transplant protocols is high in refractory Crohn's disease; this study used low doses of cyclophosphamide. Fourteen patients were submitted to non-myeloablative autologous hematopoietic stem cell transplantation. RESULTS: The average number of days of anemia (hemoglobin < 10 g/dL) was 5.4 ± 4.2 and 14 ± 2.4 in the mobilization and conditioning phases, respectively. The mean number of days of neutropenia (neutrophils < 0.5 × 109/L) in the mobilization phase was 1.7 ± 1.5 while it was 7.6 ± 1.4 in the conditioning phase. When comparing the conditioning and mobilization phases, there was an increased number days of leukopenia (white blood cells < 1.0 × 109/L), lymphocytopenia (lymphocytes < 0.5 × 109/L) and thrombocytopenia (platelets < 25 × 109/L). Crohn's Disease Activity Index values before the transplant ranged from 155 to 450.5 (mean 281.2 ± 79.0) and at 30 days after the procedures they ranged from 45.4 to 177 (mean 95.8 ± 35.4). Moreover, the procedure improved in overall quality of life of patients. Non-myeloablative autologous hematopoietic stem cell transplantation with lower doses of cyclophosphamide leads to lower rates of hematological toxicity and adverse events compared to protocols described in the literature. Trial registration NCT 03000296: Date 9 December 2016.
