Salvage haploidentical allogeneic hematopoietic stem cell transplantation using posttransplant cyclophosphamide for engraftment failure

JRCT ID: jRCTs031220607

Registered date:02/02/2023

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: engraftment failure after allogeneic hematopoietic stem cell transplantation
• Date of first enrollment: 05/07/2023
• Target sample size: 20
• Study type: Interventional
• Intervention(s): Conditioning with fludarabine of 90 mg/m2, melphalan of 40mg/m2, and 2 Gy total body irradiation, followed by salvage hematopoietic stem cell transplantation by haploidentical related donor

Outcome(s)

• Primary Outcome: Overall survival at 1 year

Secondary Outcome

1. neutrophil and platelet engraftment rate at 30 days
2. incidence and severity of acute and chronic GVHD at 180 and 365 days
3. relapse rate at 180 and 365 days
4. nonrelapse mortality at 180 and 365 days
5. relapse-free survival at 180 and 365 days
6. GVHD-free relapse-free survival at 180 and 365 days
7. incidence of infection at 180 days
8. incidence of non-hematological toxicity graded>=3 at 100 days

Key inclusion & exclusion criteria

• Age minimum: Not applicable
• Age maximum: <= 70age old
• Gender: Both

Include criteria

1. primary engraftment failure after allogeneic hematopoietic stem cell transplantation for hematological disease, primary immunodeficiency, and congenital metabolic disorder
2. any stage and disease status
3. Age <= 70
4. PS 0-3
5. any history of autologous or allogeneic stem cell transplantation
6. no evidence of severe organ dysfunction (ejection fraction >=50, total bilirubin <=2.0, CCr >=30, SpO2 >=90% (O2 <=4L/min)
7. consent to join this study
8. a HLA haploidentical donor is available

Exclude criteria

1. positive anti-HIV antibody
2. active cancer
3. need for vasopressor
4. pregnancy
5. allergic to cyclophosphamide, melphalan, fludarabine, tacrolimus, and mycophenolate Mofetil
6. need for chemotherapy before protocol therapy
7. concomitant relapse of primary malignancy
8. recurrent engraftment failure
9. presence of donor-specific anti-HLA antibody
10. judgment by physician

Related Information

• Primary Sponsor: Onizuka Makoto
• Secondary Sponsor: Harada Kaito
• Source(s) of Monetary Support: The Japanese Society for Transplantation and Cellular Therapy