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Updated: Sep 20, 2026
Intrafemoral Injection of Human Hematopoietic Stem and Progenitor Cells into Immunocompromised Mice
Published on: December 8, 2023
Hematopoietic stem cell transplantation for severe Crohn's disease
R M Craig1, A Traynor, Y Oyama
1Division of Immunotherapy, Northwestern University Medical Center, Chicago, IL 60611, USA.
Insights
Severe Crohn's disease patients unresponsive to standard therapies achieved remission with intense immune suppression and autologous hematopoietic stem cell transplantation (HSCT). This novel approach offers hope for refractory Crohn's disease cases.
Area of Science:
- Gastroenterology
- Immunology
- Hematology
Background:
- Standard therapies for severe Crohn's disease (CD), including anti-Tumor Necrosis Factor alpha (TNFalpha) antibodies, are ineffective for some patients.
- Refractory CD necessitates exploring alternative treatment strategies to achieve clinical remission and improve patient outcomes.
Observation:
- A cohort of four patients with severe, refractory CD underwent a unique treatment protocol.
- This protocol involved intense immune suppression followed by autologous hematopoietic stem cell transplantation (HSCT).
Findings:
- All four patients achieved clinical remission, evidenced by a significant reduction in the Crohn's Disease Activity Index (CDAI).
- Patients experienced resolution of diarrhea and abdominal pain, discontinuing all CD-specific therapies.
- Minor laboratory abnormalities and mild colonic inflammation persisted up to one year post-transplant, with no significant adverse events related to HSCT.
Implications:
- Autologous hematopoietic stem cell transplantation (HSCT) presents a viable therapeutic option for patients with severe Crohn's disease refractory to conventional treatments.
- Long-term follow-up is crucial to ascertain the durability of clinical remission induced by HSCT in CD patients.
Abstract:
It is clear that some patients with severe Crohn's disease (CD) fail to respond favorably to the standard treatment, including antibody to Tumor Necrosis Factor alpha (TNFalpha), We have embarked on a unique therapy for this group of patients, intense immune suppression followed by autologous hematopoietic stem cell transplantation (HSCT). The response to this approach in our first four patients has been excellent, with there being no significant untoward event from the transplantation and with each patient entering clinical remission in terms of the Crohn's Disease Activity Index off all therapy for CD and no diarrhea or abdominal pain. However, some evidence of minor laboratory abnormalities and slight inflammation of the colon on colonoscopic evaluation persist up to 1 year post-transplant. It is suggested that HSCT should be considered a reasonable option for patients who have failed standard CD therapy, although long-term follow-up will be necessary to confirm the duration of the induced clinical remission.
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