Microbiome Alterations in IL10RA-deficient Patients After HSCT

NCT ID: NCT03752372

Last Updated: 2020-08-12

Basic Information

• Recruitment Status: TERMINATED
• Total Enrollment: 13 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2017-10-01
• Study Completion Date: 2020-02-28

Brief Summary

To elucidate the longitudinal development of intestinal microbiota in patients with IL10RA deficiency after hematopoietic stem cell transplantation (HSCT). The investigators planned to collect fecal samples from IL10RA-deficient patients who received HSCT. Samples were collected more than once every three days after engraftment in lamina flow ward and at least once a week before discharge. Microbial DNA was extracted from the fecal samples. And all analysis was based on the next generation sequencing data.

Detailed Description

Hematopoietic stem cell transplantation is considered the only curative therapy for patients with interleukin-10 receptor-A(IL10RA) deficiency. The investigators aimed to collect the fecal samples of these patients throughout the conditioning, transplantation until discharge, providing a dense insight into the longitudinal development of intestinal microbiota.

Conditions

Inflammatory Bowel Diseases; Microbiota

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

HSCT cohort

IL10RA-deficient patients who received hematopoietic stem cell transplantation

hematopoietic stem cell transplantation

Intervention Type: PROCEDURE

1. Reduced intensity conditioning(RIC) chemotherapy regimen before transplantation

2. All patients were cared for in single rooms ventilated with a highly effective particulate air filtration system. All patients received intravenous immunoglobulin and antimicrobial prophylaxis, which included antiviral, antifungal, and Pneumocystis jirovecii prophylaxis with ganciclovir and micafungin, as per routine clinical practice before transplantation.

3. Umbilical cord blood transplantation

Interventions

hematopoietic stem cell transplantation

1. Reduced intensity conditioning(RIC) chemotherapy regimen before transplantation

2. All patients were cared for in single rooms ventilated with a highly effective particulate air filtration system. All patients received intravenous immunoglobulin and antimicrobial prophylaxis, which included antiviral, antifungal, and Pneumocystis jirovecii prophylaxis with ganciclovir and micafungin, as per routine clinical practice before transplantation.

3. Umbilical cord blood transplantation

Intervention Type: PROCEDURE

Eligibility Criteria

Inclusion Criteria

• IL10RA gene mutations;
• Eligible for hematopoietic stem cell transplantation.

Exclusion Criteria

• Unwilling to participate.

• Minimum Eligible Age: 1 Day
• Maximum Eligible Age: 6 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Children's Hospital of Fudan University

OTHER

Sponsor Role: lead

Responsible Party

Ying HUANG

The director of gastroenterology of Children's Hospital of Fudan University

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Ying Huang, MD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital of Fudan University

Locations

Children's hospital of Fudan university

Shanghai, Shanghai Municipality, China

Countries

China

References

Peng K, Qian X, Huang Z, Lu J, Wang Y, Zhou Y, Wang H, Wu B, Wang Y, Chen L, Zhai X, Huang Y. Umbilical Cord Blood Transplantation Corrects Very Early-Onset Inflammatory Bowel Disease in Chinese Patients With IL10RA-Associated Immune Deficiency. Inflamm Bowel Dis. 2018 Jun 8;24(7):1416-1427. doi: 10.1093/ibd/izy028.

Reference Type: RESULT

PMID: 29788474 (View on PubMed)

Other Identifiers

HSCT microbiomte

Identifier Type: -

Identifier Source: org_study_id