Hydrogen in Patients With Corticosteroid-Refractory/Dependent Chronic Graft-Versus-Host-Disease: A Single-Arm,
Liren Qian1, Miao Liu2, Jianliang Shen1
1Department of Hematology, The Sixth Medical Center, Chinese PLA General Hospital, Beijing, China.
Insights
Hydrogen-rich water shows promise for treating chronic graft-versus-host-disease (cGVHD) in patients refractory to standard therapies. This study found significant objective responses and improved survival rates with minimal side effects.
Area of Science:
- Hematology
- Immunology
- Medical Research
Background:
- Chronic graft-versus-host-disease (cGVHD) is a major cause of mortality post-allogeneic hematopoietic stem cell transplantation (HSCT).
- Current treatment options are limited for patients with cGVHD who are refractory or dependent on corticosteroids.
- Novel therapeutic strategies with favorable safety profiles are urgently needed.
Purpose of the Study:
- To investigate the therapeutic efficacy and safety of hydrogen in patients with cGVHD.
- To evaluate the objective response rate and survival outcomes in cGVHD patients treated with hydrogen-rich water.
- To assess the long-term toxicity associated with hydrogen administration.
Main Methods:
- A prospective, open-label, phase 2 clinical study was conducted.
- Patients received hydrogen-rich water orally at 4ml/kg, three times daily.
- Responses were assessed across multiple organ systems (skin, mouth, GI, liver, eyes, lungs, joints, fascia) every 3 months.
Main Results:
- 18 out of 24 enrolled patients (75%) achieved an objective response.
- No significant treatment-related toxicity was observed during the study.
- The estimated 4-year overall survival rate was 74.7%, with significantly prolonged survival in the response group (86.6% vs 0% in non-responders).
Conclusions:
- Hydrogen demonstrates significant efficacy in treating cGVHD patients, particularly those resistant to corticosteroids.
- Long-term administration of hydrogen appears safe and is not associated with significant adverse effects.
- Hydrogen therapy represents a promising, well-tolerated treatment option for refractory cGVHD.
Abstract:
Chronic graft-versus-host-disease (cGVHD) is the leading cause of late non-relapse mortality after allogeneic hematopoietic stem cell transplantation(HSCT). There is no standard therapy for patients refractory or dependent to corticosteroid treatment. We hypothesized that hydrogen may exert therapeutic effects on cGVHD patients with few side effects. A prospective open-label phase 2 study of hydrogen was conducted. Patients received hydrogen-rich water 4ml/kg orally three times a day. Responses were graded in the skin, mouth, Gastrointestinal(GI), liver, eyes, lungs and joints and fascia every 3 months. A total of 24 patients (median age 27) were enrolled. Of the 24 patients, 18 (75%; 95% CI, 55.1% to 88%) had an objective response. No significant toxicity was observed. The estimated 4-year overall survival rate was 74.7%(95% CI, 54.9%-94.5%). The survival time was significantly prolonged in the response group. The survival rate at 4 years in the response group is significantly higher than the nonresponse group (86.6% vs 0%; p= 0.000132). Hydrogen showed great efficacy on cGVHD patients and long-term administration of hydrogen was not associated with significant toxic effects. The trial was registered at www.ClinicalTrials.Gov, NCT02918188.
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