Is it time to use hematopoietic stem cell transplantation for severe and refractory crohn's disease?

Ruiz, Milton Artur; Kaiser, Roberto Luiz; Quadros, Luiz Gustavo de

doi:10.1016/j.htct.2019.06.002

Cited by 1 publication

(2 citation statements)

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“…It is well known that autologous HSCT provided treatment-free remission in a large number of patients, but the majority persisted with histological evidence of disease. A pilot study of non-myeloablative allogeneic HSCT reported the data of nine CD patients after five years of follow-up[ 53 ]. In three patients, unselected matched sibling PBSCs were used and umbilical cord blood was used in six patients.…”

Section: Discussionmentioning

confidence: 99%

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Medical, ethical, and legal aspects of hematopoietic stem cell transplantation for Crohn’s disease in Brazil

Ruiz¹,

Kaiser²,

Piron-Ruiz³

et al. 2020

WJSC

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Crohn's disease (CD) is a chronic inflammatory bowel disease that can affect any part of the gastrointestinal tract. The etiology of CD is unknown; however, genetic, epigenetic, environmental, and lifestyle factors could play an essential role in the onset and establishment of the disease. CD results from immune dysregulation due to loss of the healthy symbiotic relationship between host and intestinal flora and or its antigens. It affects both sexes equally with a male to female ratio of 1.0, and its onset can occur at any age, but the diagnosis is most commonly observed in the range of 20 to 40 years of age. CD diminishes quality of life, interferes with social activities, traumatizes due to the stigma of incontinence, fistulae, strictures, and colostomies, and in severe cases, affects survival when compared to the general population. Symptoms fluctuate between periods of remission and activity in which complications such as fistulas, strictures, and the need for bowel resection, surgery, and colostomy implantation make up the most severe aspects of the disease. CD can be progressive and the complications recurrent despite treatment with anti-inflammatory drugs, corticosteroids, immunosuppressants, and biological agents. However, over time many patients become refractory without treatment alternatives, and in this scenario, hematopoietic stem cell transplantation (HSCT) has emerged as a potential treatment option. The rationale for the use of HSCT for CD is anchored in animal studies and human clinical trials where HSCT could reset a patient's immune system by eliminating disease-causing effector cells and upon immune recovery increase regulatory and suppressive immune cells. Autologous HSCT using a non-myeloablative regimen of cyclophosphamide and anti-thymocyte globulin without CD34+ selection has been to date the most common transplant conditioning regimen adopted. In this review we will address the current situation regarding CD treatment with HSCT and emphasize the medical, ethical, and legal aspects that permeate the procedure in Brazil.

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Section: Discussionmentioning

confidence: 99%

“…Curiously, of five allogeneic UCB recipients, none had GVHD, and none had CD3+ and CD33+ donor engraftment after six months. Thus, the door is open to allogeneic UCB HSCT for CD[ 53 ].…”

Section: Discussionmentioning

confidence: 99%