Clinical trial NCT02240992: MSCs With or Without Peripheral Blood Stem Cell for Treatment of Poor Graft Function and Delayed Platelet Engraftment
Official title: Mesenchymal Stem Cells With or Without G-CSF Mobilized Peripheral Blood Stem Cell for Treatment of Poor Graft Function and Delayed Platelet Engraftment After Allogeneic Hematopoietic Stem Cell Transplant
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
PHASE3
Conditions: Stem Cell Transplantation, Hematopoietic
Poor Graft Function
Delayed Platelet Engraftment
Hematological Diseases
Keywords: Poor Graft Function
Delayed platelet engraftment
Peripheral Blood Stem Cell
Mesenchymal Stem Cells
Allogeneic hematopoietic stem cell transplantation
Lead sponsor: Nanfang Hospital, Southern Medical University
Sponsor class: OTHER
Collaborators: name: Guangdong Provincial People's Hospital
name: Third Affiliated Hospital, Sun Yat-Sen University
name: Guangzhou General Hospital of Guangzhou Military Command
name: Guangzhou First People's Hospital
name: Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
name: Southern Medical University, China
name: Peking University People's Hospital
name: Huazhong University of Science and Technology
name: Sun Yat-sen University
name: Academy Military Medical Science, China
First posted: 2014-09-16
Enrollment: 120
Sex: ALL
Minimum age (years): 14
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to compare the efficacy of mesenchymal stem cells (MSCs) with or without granulocyte colony-stimulating factor (G-CSF) mobilized peripheral stem cells (PBSC) in treating patients experiencing poor graft function or delayed platelet engraftment after allogeneic hematopoietic stem cell transplantation.

Detailed description: Allogeneic hematopoietic stem cell transplantation(allo-HSCT) is the only cure for many hematologic diseases. However, about 5-27% of patients would suffer from poor graft function (PGF) and more recipients might develop delayed platelet engraftment (DPE) after allo-HSCT. These complications are associated with considerable mortality related to infections or hemorrhagic complications. Treatment of PGF and DPE usually involves hematopoietic growth factors such as granulocyte colony-stimulating factor (G-CSF) and thrombopoietin (TPO), or second transplantation, but these methods have dismal effect or even a significant risk of graft-versus-host disease (GVHD).

Mesenchymal stem cells (MSCs) are a form of multipotent adult stem cells that can be isolated from bone marrow (BM), adipose tissue, and cord blood. Clinical applications of human MSCs include improving hematopoietic engraftment, preventing and treating graft-versus-host disease after allo-HSCT and so on. Some studies have shown that MSCs combined with PBSC or cord blood could be useful to improve engraftment after HSCT. Several reports suggested MSCs might be effective in the treatment of PGF.

However, the efficacy of MSCs as single-drug treatment for PGF or DPE is unsatisfactory in our previous study. Therefore, in the present study, G-CSF mobilized PBSC will be used combined with MSCs in the patients with PGF or DPE after allo-HSCT.

Eligibility criteria: Inclusion Criteria:

* Age：14-65 years
* PGF or DPE after allo-HSCT
* Subjects (or their legally acceptable representatives) must have signed an informed consent document

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: Percentage of Participants with Hematopoietic Recovery
timeFrame: 1 year
Secondary outcomes:
measure: Incidence of graft-versus-host disease
timeFrame: 1 year
measure: Incidence of infections
timeFrame: 1 year
measure: Incidence of primary underlying disease relapse
timeFrame: 1 year
measure: Incidence of acute toxicity
timeFrame: up to 100 days
Other outcomes:
Locations:
city: Guangzhou
country: China
facility: Department of Hematology,Nanfang Hospital, Southern Medical University
state: Guangdong
Study officials:
affiliation: Nanfang Hospital, Southern Medical University
name: Qifa Liu, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Sanchez-Guijo FM, Lopez-Villar O, Lopez-Anglada L, Villaron EM, Muntion S, Diez-Campelo M, Perez-Simon JA, San Miguel JF, Caballero D, del Canizo MC. Allogeneic mesenchymal stem cell therapy for refractory cytopenias after hematopoietic stem cell transplantation. Transfusion. 2012 May;52(5):1086-91. doi: 10.1111/j.1537-2995.2011.03400.x. Epub 2011 Oct 24.
citation: Meuleman N, Tondreau T, Ahmad I, Kwan J, Crokaert F, Delforge A, Dorval C, Martiat P, Lewalle P, Lagneaux L, Bron D. Infusion of mesenchymal stromal cells can aid hematopoietic recovery following allogeneic hematopoietic stem cell myeloablative transplant: a pilot study. Stem Cells Dev. 2009 Nov;18(9):1247-52. doi: 10.1089/scd.2009.0029.