Effects of Mesenchymal Stem Cell Supernatant on Prevention and Treatment of Skin/Mucosal Injury in Hematology Patients

This clinical trial aims to evaluate the efficacy and safety of mesenchymal stem cell (MSC) culture supernatant in the prevention and treatment of mucosal injuries in patients undergoing hematopoietic stem cell transplantation (HSCT). HSCT is a critical therapy for blood-related cancers and severe hematological disorders but is often associated with significant mucosal injuries caused by the high-dose chemotherapy and radiation therapy used during the preconditioning phase. These injuries predominantly affect the oral mucosa, skin, and bladder, resulting in severe pain, elevated infection risks, and prolonged hospitalization.

Current treatment options for mucosal injuries focus on symptom management but provide limited efficacy in accelerating tissue repair or preventing further complications. MSC culture supernatant contains a variety of bioactive factors, including growth factors and cytokines secreted by MSCs during culture. These factors are believed to promote tissue repair and regeneration, offering a promising alternative to live cell therapies, which pose higher safety risks. This study investigates MSC supernatant as a novel therapeutic option for mucosal injuries in HSCT patients.

Study Design:

This is a prospective, randomized controlled trial with a target enrollment of 120 patients. Participants will be randomly assigned to one of two groups:

Control Group: Patients will receive standard care for mucosal injuries, which may include oral rinses, pain management, and supportive care.

Intervention Group: Patients will receive MSC culture supernatant in addition to standard care. The MSC supernatant will be administered based on the type of mucosal injury:

Oral Mucosal Injuries: 15 mL of MSC supernatant will be used as a mouthwash. Skin Mucosal Injuries: MSC supernatant will be applied topically, proportional to the affected body surface area.

Bladder Injuries (Cystitis): 50 mL of MSC supernatant will be used for bladder irrigation.

Study Objectives:

Primary Objective: To assess the efficacy of MSC culture supernatant in the healing of mucosal injuries in HSCT patients. Mucosal healing will be evaluated using standardized scales such as the WHO mucosal injury scale and patient-reported pain scores.

Secondary Objective: To determine the safety and tolerability of MSC supernatant by monitoring adverse events (AEs) and serious adverse events (SAEs) in accordance with the CTCAE v5.0 criteria.

Exploratory Objectives: To explore the impact of MSC supernatant on tissue repair, infection reduction, length of hospital stay, and patient quality of life (QoL) during recovery from HSCT.

Inclusion and Exclusion Criteria:

Inclusion criteria

Patients who have undergone HSCT and developed mucosal injuries (oral, skin, or bladder).

Ability to understand and provide informed consent, and comply with study requirements.

Exclusion criteria

Patients with severe organ dysfunction or uncontrolled active infections. Patients with known allergies to MSC supernatant. Pregnant or breastfeeding women. Patients who are HIV-positive or have active hepatitis B or C. Patients enrolled in another clinical trial within the last four weeks.

Outcome Measures:

Primary Outcome: Mucosal injury healing, defined as a reduction in the mucosal injury grade to ≤1 and a pain score of ≤2.

Secondary Outcomes: Frequency and severity of adverse events related to MSC supernatant, measured according to CTCAE v5.0.

Exploratory Outcomes: Reduction in infection rates, shortened hospital stays, and improved patient-reported quality of life (QoL).

Monitoring and Safety:

Participants will undergo regular evaluations, including weekly assessments during the treatment phase and monthly follow-ups post-treatment. Laboratory tests (e.g., blood counts, liver and kidney function tests) and vital signs will be closely monitored to ensure patient safety. Adverse events will be documented, and appropriate interventions will be implemented as required.

It is important to note that while no Data Monitoring Committee (DMC) has been established for this study, safety oversight will be conducted by the research team following standard safety monitoring protocols.

Study Duration:

The trial is set to run from October 2020 to September 2025, with data collection for the primary outcomes expected to be completed by September 2025. Participants will be followed for the entirety of the study period, and long-term assessments will be conducted to evaluate the sustained efficacy and safety of MSC supernatant for mucosal injuries.

Significance:

This trial has the potential to introduce an innovative treatment strategy for managing mucosal injuries in HSCT patients. By utilizing the bioactive components of MSC culture supernatant, this therapy may enhance tissue regeneration, lower infection risk, and expedite recovery. If proven effective, this treatment could significantly improve patient outcomes and overall quality of life during and after HSCT.

Inclusion criteria

• Participants aged 18 years or older.
• Patients who have undergone hematopoietic stem cell transplantation (HSCT).
• Patients who have developed mucosal injuries (oral, skin, or bladder) after HSCT, chemotherapy, or radiotherapy.
• Patients who are willing to provide informed consent and comply with the study procedures.

Exclusion criteria

• Patients with severe organ dysfunction (e.g., heart, liver, kidney failure) that could interfere with the study.
• Patients with uncontrolled active infections.
• Known allergies or hypersensitivity to MSC supernatant.
• Pregnant or breastfeeding women.
• Patients with HIV or active hepatitis B or C infections.
• Patients who have participated in another clinical trial within the last 4 weeks.