Clinical trial NCT03756675: Haploidentical Peripheral Blood Stem Cell Transplantation for Acute Leukemia
Official title: Haploidentical Peripheral Blood Stem Cell Transplantation for Acute Leukemia
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Acute Leukemia
Keywords: allogeneic stem cell transplantation
G-CSF-mobilized peripheral blood stem cell
haplotype
Lead sponsor: Peking University People's Hospital
Sponsor class: OTHER
First posted: 2018-11-28
Enrollment: 45
Sex: ALL
Minimum age (years): 2
Maximum age (years): 60
Standard ages: CHILD
ADULT
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients aged 2-60, who plan to receive haplotype PBSCT in the first complete remission phase (CR1) of acute leukemia, and with no uncontrolled current infections or organ failure.

Brief summary: Allogeneic stem cell transplantation (Allo-HSCT) is the effective and even the only treatment for hematological malignancies. The "GIAC" protocol established by our center has successfully crossed the HLA barrier in HLA-mismatched/haploidentical HSCT. The protocol entails the following: treating donors with granulocyte colony-stimulating factor (G-CSF) to induce donor immune tolerance, intensified immunologic suppression to both promote engraftment and to prevent GVHD, antithymocyte globulin (ATG) was included for the prophylaxis of GVHD and graft rejection, and combination of G-CSF-primed bone marrow harvest (G-BM) and G-CSF-mobilized peripheral blood stem cell harvest (G-PB) as the source of stem cell grafts. But peripheral blood transplantation is still prevalent. Compared with BM, G-PB is more convenient to collect, and the number of T lymphocytes and CD34+ cells is higher. It is reported that G-PB has a higher implantation rate and even a higher disease-free survival rate in sibiling-identical transplantation compared with BM transplantation, whereas there were also reports with different conclusions. This prospective, one-arm clinical cohort study aims to evaluate the safety and efficacy of haplotype peripheral blood stem cell transplantation (PBSCT) in the treatment of acute leukemia.

Detailed description: Allogeneic stem cell transplantation (Allo-HSCT) is the effective and even the only treatment for hematological malignancies. The "GIAC" protocol established by our center has successfully crossed the HLA barrier in HLA-mismatched/haploidentical HSCT. The protocol entails the following: treating donors with granulocyte colony-stimulating factor (G-CSF) to induce donor immune tolerance, intensified immunologic suppression to both promote engraftment and to prevent GVHD, antithymocyte globulin (ATG) was included for the prophylaxis of GVHD and graft rejection, and combination of G-CSF-primed bone marrow harvest (G-BM) and G-CSF-mobilized peripheral blood stem cell harvest (G-PB) as the source of stem cell grafts. But peripheral blood transplantation is still prevalent. Compared with BM, G-PB is more convenient to collect, and the number of T lymphocytes and CD34+ cells is higher. It is reported that G-PB has a higher implantation rate and even a higher disease-free survival rate in sibiling-identical transplantation compared with BM transplantation, whereas there were also reports with different conclusions. This prospective, one-arm clinical cohort study aims to evaluate the safety and efficacy of haplotype peripheral blood stem cell transplantation (PBSCT) in the treatment of acute leukemia.

Eligibility criteria: Inclusion Criteria:

* 2-60 years old, all genders;
* the first complete remission phase (CR1) of acute leukemia;
* planning to receive haplotype PBSCT;
* no uncontrolled current infections (new infections, body temperature still above 38 ℃ after treatment with broad-spectrum antibiotics for 72h, except for other non-infectious factors);
* no organ failure.

Exclusion Criteria:

* with poor compliance;
* with uncontrolled current infections；
* pregnancy;
* donors with contraindications of mobilization and collection of peripheral blood stem cells;
* with mental sickness

Primary outcomes:
measure: engraftment rate
timeFrame: one year after transplantation
Secondary outcomes:
measure: cumulative incidence of acute graft-versus-host disease(GVHD)
timeFrame: one year after transplantation
measure: cumulative incidence of chronic GVHD at one year
timeFrame: one year after transplantation
measure: cumulative incidence of relapse at one year
timeFrame: one year after transplantation
measure: cumulative incidence of non-relapse mortality (NRM) at one year
timeFrame: one year after transplantation
measure: overall survival at one year
timeFrame: one year after transplantation
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking University People's Hospital
state: Beijing
Study officials:
affiliation: Peking University People's Hospital
name: Xiao-Jun Huang, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Shen MZ, Hong SD, Lou R, Chen RZ, Zhang XH, Xu LP, Wang Y, Yan CH, Chen H, Chen YH, Han W, Wang FR, Wang JZ, Liu KY, Huang XJ, Mo XD. A comprehensive model to predict severe acute graft-versus-host disease in acute leukemia patients after haploidentical hematopoietic stem cell transplantation. Exp Hematol Oncol. 2022 May 3;11(1):25. doi: 10.1186/s40164-022-00278-x.
citation: Shen MZ, Hong SD, Wang J, Zhang XH, Xu LP, Wang Y, Yan CH, Chen H, Chen YH, Han W, Wang FR, Wang JZ, Liu KY, Huang XJ, Mo XD. A Predicted Model for Refractory/Recurrent Cytomegalovirus Infection in Acute Leukemia Patients After Haploidentical Hematopoietic Stem Cell Transplantation. Front Cell Infect Microbiol. 2022 Mar 22;12:862526. doi: 10.3389/fcimb.2022.862526. eCollection 2022.
citation: Ma YR, Zhang X, Xu L, Wang Y, Yan C, Chen H, Chen Y, Han W, Wang F, Wang J, Liu K, Huang X, Mo X. G-CSF-Primed Peripheral Blood Stem Cell Haploidentical Transplantation Could Achieve Satisfactory Clinical Outcomes for Acute Leukemia Patients in the First Complete Remission: A Registered Study. Front Oncol. 2021 Mar 15;11:631625. doi: 10.3389/fonc.2021.631625. eCollection 2021.