Cord Blood Transplant Corrects Very Early Onset Irritable Bowel Disease

By Braydeen Tank 5 days ago
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Cord Blood Transplant Corrects Very Early Onset Irritable Bowel Disease

After only 8 days after her birth, Baby Zhao began to suffer from a fever and severe diarrhoea. As her symptoms only grew worse her parents sought out help at the Children’s Hospital of Fudan University in Shanghai. They diagnosed her with Very Early Onset Irritable Bowel (VEO-IBD) with IL10RA immune deficiency. This causes severe dysregulation of the immune system in the intestines which leads to severe malnourishment and often abscesses and fistulas in the intestines.

The only known treatment of this type of VEO-IBD is a stem cell transplant. Baby Zhao was the first of 9 patients at the children’s hospital to receive a cord blood transplant for VEO-IBD with IL10RA immune deficiency. Within one to three months after the treatment, baby Zhao and the other patients were experiencing normal stools. When baby Zhao got home after her stem cell transplant her mother commented that she “remember(s) when she was 100 days old, she was skinny like a skeleton. Now look at her: She’s almost got a puffy face.”

After only 8 days following her birth, baby Zhao began to suffer from a fever and diarrhoea. Zhao’s parents had given birth to baby girl, before Zhao, who had suffered from the same symptoms within her first week of life; and after only 5 months she passed away as a result. To prevent the same fate for their new baby girl, when she began to show symptoms her parents took her to many local clinics and when none could help, they went to the Children’s Hospital of Fudan University in Shanghai.

The doctors at the children’s hospital eventually diagnosed Zhao with Very Early Onset Irritable Bowel Disease (VEOIBD) with IL10RA immune deficiency, which is a form of the disease caused an inherited genetic mutation in the IL10RA gene. This type of VEOIBD causes severe dysregulation of the immune system in the intestines which leads to severe malnourishment and often abscesses and fistulas in the intestines.

Irritable Bowel Syndrome in children is different to IBS in adults as it is often resistant to many of the standard medications used for treatment. According to Huang Ting, MD PHD, who is the director of the digestive disease department at the Children’s hospital, “the only effective treatment (for VEOIBD) is a (haematopoietic) stem cell transplant” as “it can help repair the genetic deforming and control the symptoms of the disease.”

Zhao was the first patient to receive a cord blood transplant at the hospital for her VEO-IBD with IL10RA immune deficiency, of which 8 more patients received transplants over the next 2 years. They performed the transplants in conjunction with a low intensity chemotherapy (to minimise the toxicity in infants who already have malnutrition and infections in their intestines). Within 1 to 3 months after the transplants, the 9 patients including baby Zhao, experienced normal stools. When baby Zhao got home after her stem cell transplant her mother commented that she “remember(s) when she was 100 days old, she was skinny like a skeleton. Now look at her: She’s almost got a puffy face.”

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