Clinical trial NCT05667402: Clinical Study of TBF Regimen in Allo-HSCT in Patients With CNS Leukemia
Official title: Clinical Study of TBF Regimen in Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Central Nervous System Leukemia
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: NA
Conditions: Central Nervous System Leukemia
Allogeneic Hematopoietic Stem Cell Transplantation
Lead sponsor: First Affiliated Hospital Xi'an Jiaotong University
Sponsor class: OTHER
First posted: 2022-12-28
Enrollment: 50
Sex: ALL
Minimum age (years): 14
Maximum age (years): 60
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: Central nervous system (CNS) leukemia is a poor prognostic factor for allogeneic hematopoietic stem cell transplantation (allo-HSCT). Thiotepa can penetrate the blood-brain barrier and has immunosuppressive effects and similar effects to irradiation in allo-HSCT. This project aims to investigate whether the TBF regimen is superior to the traditional modified BuCY2 regimen to improve the long-term survival of the CNS leukemia patients.

Detailed description: Central nervous system (CNS) leukemia is a common extramedullary leukemia and a poor prognostic factor for allogeneic hematopoietic stem cell transplantation (allo-HSCT). There is a blood-brain barrier (BBB) in the CNS. The treatment effect of CNS leukemia is seriously limited by the low penetration of conventional chemotherapy drugs into cerebrospinal fluid. Thiotepa can penetrate the BBB and has immunosuppressive effect and myeloablative effect similar to irradiation in transplantation. Therefore, it is speculated that cestipide has certain advantages as a conditioning regimen in transplantation for CNS leukemia. This project aims to investigate whether the TBF regimen is superior to the traditional modified BuCY2 regimen to improve the long-term survival of the CNS leukemia patients.

Eligibility criteria: Inclusion Criteria:

* Patients diagnosed with central nervous system leukemia by MICM meet one of the following conditions: corresponding symptoms and signs of central nervous system involvement; cerebrospinal fluid pressure increased by 200 mm water column; the white blood cell count in cerebrospinal fluid was 0.01×10\^9/L; cerebrospinal fluid protein qualitative test was positive or protein quantification was 45 mg/dL; leukemic cells can be found in the cerebrospinal fluid; cranial imaging suggested central involvement.
* Aged 14-60 years, male or female.
* KPS score: ≥80.
* Signed the informed consent.

Exclusion Criteria:

* Patients intending to receive autologous hematopoietic stem cell transplantation.
* Patients with transplantation contraindications.
* Those who refuse to sign the informed consent form.

Primary outcomes:
measure: Recurrence rate of central nervous system (CNS) leukemia
timeFrame: From date of the treatment with the conditioning regimen until CNS leukemia relapse, the end of follow-up or the date of death from any cause, whichever came first, assessed up to 36 months.
Secondary outcomes:
measure: Hematopoietic implantation rate
timeFrame: From date of the treatment with the conditioning regimen until hematopoietic implantation, assessed up to 100 days..
measure: NRM
timeFrame: From date of allo-HSCT until the date of death from any cause or the end of follow-up, whichever came first, assessed up to 36 months.
measure: OS
timeFrame: From date of the treatment with the conditioning regimen until the end of follow-up or the date of death from any cause, whichever came first, assessed up to 36 months.
measure: PFS
timeFrame: From date of the treatment with the conditioning regimen until leukemia progression, the end of follow-up or the date of death from any cause, whichever came first, assessed up to 36 months.
measure: AEs
timeFrame: From date of the treatment with the conditioning regimen until the occurrence of adverse effects, the end of follow-up or the date of death from any cause, whichever came first, assessed up to 36 months.
Other outcomes:
Locations:
city: Xi'an
country: China
facility: First Affiliated Hospital of Xi'an Jiaotong University
state: Shaanxi
Study officials:
affiliation: First Affiliated Hospital of Xian Jiaotong University
name: Pengcheng He, MD
role: PRINCIPAL_INVESTIGATOR
References: