Clinical trial NCT06650553: Umbilical Cord Blood-Supported Haplo-HSCT for Aplastic Anemia Treatment Study
Official title: Clinical Study on Umbilical Cord Blood-Supported Haploidentical Hematopoietic Stem Cell Transplantation for the Treatment of Aplastic Anemia
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Aplastic Anemias
Keywords: Aplastic Anemias
Stem Cell Transplantation
Umbilical Cord Blood
Lead sponsor: Shanxi Bethune Hospital
Sponsor class: OTHER
First posted: 2024-10-21
Enrollment: 110
Sex: ALL
Minimum age (years): 14
Maximum age (years): 70
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Aplastic anemia (AA) is a rare bone marrow failure syndrome with an annual incidence of about 0.74/100,000, affecting all ages but more common in the elderly. It's divided into congenital and acquired forms, with the latter being more prevalent. The primary acquired form is linked to T lymphocyte activation and genetic factors. The best treatment is allogeneic hematopoietic stem cell transplantation (allo-HSCT), with a near 90% cure rate. Sibling allo-HSCT is ideal but finding a match is challenging. For those who relapse after immunosuppressive therapy, haploidentical HSCT is a viable option despite risks like graft failure and GVHD. Advances in transplantation have made haplo-HSCT's efficacy comparable to other methods. Recent studies suggest co-transplantation with umbilical cord blood cells can improve outcomes by hastening hematopoietic recovery and prognosis. Our study will evaluate the feasibility and safety of this approach in AA treatment, comparing it to sibling fully matched transplantation, with a focus on infection rates, GVHD incidence, TRM, and EFS, aiming to enhance treatment practices and benefit patients and the medical industry.

Detailed description: Aplastic anemia (aplastic anaemia，AA) is a syndrome of bone marrow hematopoietic dysfunction （bone marrow hematopoietic failure，BMF）with an annual incidence rate of approximately 0.74/100,000 people, affecting all age groups with a higher incidence rate in the elderly, and similar incidence rates between males and females. AA is divided into congenital and acquired types, with acquired AA being more common. The pathogenesis of primary acquired AA is mainly related to the abnormal activation of T lymphocytes, and genetic background may also play a role in the development and progression of AA. Currently, allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the best treatment for AA, with a cure rate close to 90%. Sibling allogeneic hematopoietic stem cell transplantation (HLA-matched HSCT) is the preferred treatment plan, but it is not easy to find a fully matched sibling donor. For patients who relapse after immunosuppressive therapy (IST), haploidentical hematopoietic stem cell transplantation (haplo-HSCT) is a good alternative, despite the risks of engraftment failure, infection, and graft-versus-host disease (GVHD). With the advancement of transplantation technology, the efficacy of haplo-HSCT is not significantly different from that of unrelated donor hematopoietic stem cell transplantation (MUD-HSCT) and HLA-matched sibling donor hematopoietic stem cell transplantation (MSD-HSCT). Studies have shown that haplo-HSCT has a higher survival rate in treating patients with severe aplastic anemia (SAA), and there is no significant difference compared to MSD-HSCT.To address the risk of complications associated with haplo-HSCT, recent research has indicated that co-transplantation with third-party umbilical cord blood cells can shorten the time for hematopoietic reconstitution and improve prognosis. The co-transplantation of third-party umbilical cord blood with bone marrow and peripheral blood hematopoietic stem cells has become a new transplantation option. Studies at home and abroad have shown that combined third-party transplantation can shorten the time for hematopoietic reconstitution and improve long-term survival rates. This study plans to adopt a prospective, open-label, parallel-controlled trial design to conduct a clinical study of non-related umbilical cord blood-assisted haploidentical allogeneic hematopoietic stem cell transplantation for the treatment of AA. The study will compare the umbilical cord blood combined with haploidentical transplantation plan with sibling fully matched transplantation, to assess the feasibility and safety of the umbilical cord blood combined with haploidentical transplantation plan, and to monitor the incidence of patient infections, acute/chronic GVHD incidence, transplant-related mortality (TRM), and event-free survival (EFS), in the hope of providing new practical experience in the field of AA treatment, and bringing dual benefits to patients and the social medical enterprise.

Eligibility criteria: Inclusion Criteria:

* A patient age of 14-70 years
* Patients diagnosed with Severe Aplastic Anemia (SAA) or Transfusion-Dependent Non-Severe Aplastic Anemia (TD-NSAA) according to the Chinese Guidelines for the Diagnosis and Treatment of Aplastic Anemia (2022 Edition) and suitable for allo-HSCT
* Subjects (or their legally acceptable representatives) must have signed an informed consent document indicating that they understand the purpose of and procedures required for the study and are willing to participate in the study

Exclusion Criteria:

* Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure)
* Patients with any conditions not suitable for the trial (investigators' decision)

Primary outcomes:
measure: Time to neutrophil engraftment
timeFrame: 60 days
Secondary outcomes:
measure: Time to platelet engraftment
timeFrame: 60 days
measure: Incidence of infections (including CMV and EBV incidence)
timeFrame: 100 days,6 months,12 months,24 months
measure: Overall survival rate (OS)
timeFrame: 24 months
measure: Event-free survival (EFS)
timeFrame: 24 months
measure: Incidence of acute/chronic GvHD
timeFrame: 24 months
measure: Transplant-related mortality (TRM)
timeFrame: 100 days,6 months,12 months,24 months
Other outcomes:
Locations:
city: Taiyuan
country: China
facility: Shanxi Bethune Hospital
state: Shanxi
Study officials:
affiliation: Shanxi Bethune Hospital Regulatory Authority
name: Tao Wang, Dr.
role: STUDY_DIRECTOR
References: