Clinical trial NCT06837987: Modified Transplantation Regimen and AGVHD Prophylaxis for Severe Aplastic Anemia in the Setting of Allogeneic Hematopoietic Stem Cell Transplantation
Official title: An Observation of the Safety and Efficacy of a Modified Transplantation Conditioning and AGVHD Prophylaxis for Severe Aplastic Anemia--a Retrospective, Double-center, Single-arm Clinical Study.
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Aplastic Anaemia
Transplantation Conditioning
Keywords: modified transplantation conditioning
aplastic anemia
aGVHD prophylaxis
Lead sponsor: Hematology department of the 920th hospital
Sponsor class: OTHER
First posted: 2025-02-20
Enrollment: 52
Sex: ALL
Minimum age (years): 3
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients who were diagnosed with severe aplastic anemia by NCCN guidelines, and has received HSCT with this transplantation preconditioning and aGVHD prophylaxis regimen;

Brief summary: We retrospectively evaluted the safety and efficacy of the modified transplantation conditioning and aGVHD prophylaxis in severe aplastic anemia in two clinical center.

Detailed description: Aplastic anemia (AA) is a group of myelo-hemopoietic failure syndromes caused by a variety of etiologies. If not intervened, the average expected survival time is less than half a year.Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is one of the possible cures for this disease. The success rate of treatment for this disease can be further improved under the previous regimen system. The survival rate reported in the literature is 60%-90%. From Jun. 2020 to Jun 2023, 52 patients in two transplantation center received this modified transplantation system, we designed this clinical trial to retrospectively evaluted the safety and efficacy of the modified transplantation conditioning and aGVHD prophylaxis in severe aplastic anemia in these two clinical center.

Eligibility criteria: Inclusion Criteria:1.Patients who were diagnosed with severe aplastic anemia by NCCN guidelines, and has received HSCT with this transplantation preconditioning and aGVHD prophylaxis regimen; 2.Age 3-65 years old; 3.Weight 10Kg-100Kg; 4.Eastern Cooperative Oncology Group (ECOG) score ≤3; 5.No major organ injury (ECG ejection fraction \>45%; bilirubin \< 2 times the upper limit of normal value; AST and ALT \< 3 times the upper limit of normal value; serum creatinine \< 2 times the upper limit of normal value); 6.No severe infection; 7.Subjects voluntarily participated in this clinical trial and signed the informed consent.

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Exclusion Criteria:1.patients with other hematologic diseases who are not eligible for transplantation or who do not wish to receive transplantation; 2.patients with an expected survival of less than 1 month; 3.patients with previous autologous or allogeneic hematopoietic stem cell transplantation; 4.pregnant patients; 5.patients with severe mental or neurological disorders that would affect the ability to provide informed consent and/or to report or observe adverse events; 6.other conditions that the investigator determines to be inappropriate for enrollment.

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Primary outcomes:
measure: Primary graft falure rate
timeFrame: 24 months
measure: aGVHD incidence
timeFrame: within 100 days after HSCT
Secondary outcomes:
measure: Poor graft function
timeFrame: 24 months
measure: cGVHD incidence
timeFrame: 24 months
measure: adverse events
timeFrame: within 100 days after HSCT
measure: 2-year Overall survival rate
timeFrame: 24 months
measure: Disease-free survival
timeFrame: 24 months
measure: Transplantation-related mortality
timeFrame: 2 years
Other outcomes:
measure: Fungal infections rate
timeFrame: 2 years
measure: EB virus reactivation rate
timeFrame: within 100 days after HSCT
measure: cytomegalovirus reactivatioin
timeFrame: within 100 days after HSCT
Locations:
city: Kunming
country: China
facility: Department of Hematology，920th Hospital of Joint Logistics Support Force
state: Yunnan
Study officials:
References: