ATG Combined With Cyclophosphamide And Cord Blood Transfusion in Treating Patients With Severe Aplastic Anemia

Jinan Military General Hospital130 enrolled

Overview

To assess whether ATG Combined With Cyclophosphamide and cord blood infusion can accelerate hematopoietic reconstruction in severe aplastic anemia patients and improve clinical curative effect and safety

Aplastic Anemia( AA), is a set of bone marrow hematopoietic dysfunction caused by a variety of causes, with hyperplasia of bone marrow hematopoietic cells to reduce whole blood cells and peripheral blood at the characteristics of clinical main performance for anemia, bleeding and infection. According to the severity of the bone marrow failure and the progress of the clinical course ,it is divided into Severe Aplastic Anemia (SAA) and the Non - Severe Aplastic Anemia (NSAA).Severe Aplastic Anemia can be divided into two categories: Very Severe Aplastic Anemia (VSAA) and Severe Aplastic Anemia (SAA), with the characteristics of rapid progress, refractory, poor prognosis, high mortality .The natural course is six months or so, and most patients die in a year . Hematopoietic stem cell transplantation and immunosuppressive therapy are two main treatment . The former is by far the only possible cure. It is recommended as first-line treatments, if patients have a matched sibling donor. The recommended age limit is 40 years old. But for those who have no sibling donor or patients older than 40 years old, it is recommend the immunosuppressive therapy. The investigators have already summarized the effectiveness of rabbit antithymocyte immunoglobulin (ATG), cyclophosphamide (Cy) and cyclosporine, A (CsA) and the combination of the umbilical cord blood infusion for SAA/VSAA patients without suitable donor, with short duration, without long-term immunosuppressive therapy history. The total effectiveness rates has improved to 88%, with shorter immunosuppressive maintaining therapy , rapid hematopoietic reconstruction, fewer complications. The aim of this study is to further explore whether this solution can accelerate hematopoietic reconstruction of SAA/VSAA patients and its clinical curative effect and security. This study scheme has been approved by the Jinan military region general hospital medical ethics committee.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

130

Conditions

Aplastic Anemia

Interventions

Rabbit ATG, (Genzyme)DRUG

ATG is an infusion of rabbit-derived antibodies against human T cells, which is used in the prevention and treatment of acute rejection in organ transplantation and therapy of aplastic anemia

CyDRUG

Cyclophosphamide is a medication mainly used in chemotherapy. It is an alkylating agent of the nitrogen mustard type

CsADRUG

CsA is an immunosuppressant drug widely used in organ transplantation to prevent rejection. It reduces the activity of the immune system by interfering with the activity and growth of T cells

Eligibility

Sex: ALLMin age: 1 YearMax age: 60 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Male or female ,under the age of 60. 2. Diagnosis of SAA and VSAA in accordance with the \<aplastic anemia, diagnosis and treatment expert consensus\> Camitta standard (see appendix 1). 3. Confirmed of heavy and very heavy aplastic anemia within 6 months. 4. No obvious abnormal liver and kidney function: ALT, AST,≤2.5 times the upper limit of normal , serum Creatinine and BUN ≤1.25 times the upper limit of normal 5. Clear understanding, voluntary to participate in the study, and signed informed consent document by the patient or the legal guardian 6. Willingness and ability to comly with the treatment plan, follow-up and laboratory tests as required Exclusion Criteria: 1. Congenital aplastic anemia 2. Pregnancy or breastfeeding 3. Participated in other clinical trials within three months 4. Presence of Any fatal disease, including respiratory failure, heart failure, liver or kidney failure, et al 5. Aplastic anemia caused by the treatment of other malignant tumor treatment 6. With severe mental illness 7. With other malignant tumor 8. Severe infection or the infection difficult to be controlled 9. Received ATG or cyclosporine A within six months 10. Severely allergic to biological agents 11. Any other situation judged by the investigator that the patients inappropriate for entry into this study

Locations (1)

The General Hospital Of Jinan Military Command

Jinan, Shandong, China

Outcomes

Primary Outcomes

The total response rate

Response rate is the ratio of CR and PR patients to all evaluated patients at the time point. CR,PR,NR and relapse is evaluated according to the guidelines for the diagnosis and management of adult aplastic anaemia from British Committee for Standards in Haematology (BCSH)

Time frame: Every 3 months to 24 months

Secondary Outcomes

Neutrophil recovery time

The neutrophil recovery day is defined from day "0" until the ﬁrst of 3 consecutive days during which the absolute neutrophil count (ANC) is \>0.5×10\^9/L, without G-CSF administration .

Time frame: From day 0 until the ﬁrst of 3 consecutive days

Infection rates

Time frame: 1 year

Overall survival

Time frame: 2 years

Treatment related mortality

Time frame: 2 years

Central Contacts

Fang Zhou, MD

CONTACT

86-0531-51665316 zhoufang1@medmail.com.cn

Zhe Yu, MD

CONTACT

86-0531-51665781 doctoryu1120@163.com

Data from ClinicalTrials.gov

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