Prospective Study of Patients With Thrombocytopenia Following HSCT

Thrombocytopenia Clinical Trial

Official title:

A Prospective Study of Patients With Isolated Thrombocytopenia Following Hematopoietic Stem Cell Transplantation

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02487563
• Other study ID #: SOOCHOW-HY-2015
• Status: Completed
• Phase: Phase 3
• Start date: October 2015
• Est. completion date: April 12, 2019

Study information

• Verified date: April 2020
• Source: The First Affiliated Hospital of Soochow University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Isolated thrombocytopenia is a common and severe complication of HSCT, which often leads to an increased risk of life-threatening hemorrhage, frequent requirement of platelet transfusions and extended hospital stays, representing a challenging clinical problem. Current treatments for thrombocytopenia after HSCT are frequently unsatisfactory in platelet recovery and for preventing potentially fatal bleeding complications. Therefore, it is urgent to explore an effective therapy to improve the outcomes of thrombocytopenia after HSCT. Previous studies have demonstrated that decitabine, a hypomethylating agent, may reduce platelet transfusions in myelodysplastic syndrome (MDS) patients. The investigators conducted an prospective clinical trial to evaluate the safety and efficiency of rhTPO and decitabine in the treatment of thrombocytopenia following HSCT.

Description:

Isolated thrombocytopenia is a frequent and severe complication of hematopoietic stem cell transplantation (HSCT). It often leads to an increased risk of life-threatening hemorrhage, frequent requirement of platelet transfusions and extended hospital stays, representing a challenging clinical problem. Current treatments for thrombocytopenia after HSCT, including thrombopoietin, interleukin-11, immunoglobulin, methylprednisolone and rituximab, are frequently unsatisfactory in platelet recovery. Therefore, it is urgent to explore an effective therapy to improve the outcomes of thrombocytopenia after HSCT. Thrombopoietin (TPO) is a cytokine that drives thrombopoiesis by stimulating the differentiation of stem cells into megakaryocytes and promoting megakaryocyte proliferation and polyploidization. Decitabine was approved for the treatment of myelodysplastic syndrome (MDS) as a DNA methylation inhibitors. Studies in vitro show that decitabine enhances platelet release and megakaryocyte maturation. Here, the investigators performed a prospective clinical trial, in order to investigate the safety and efficiency of rhTPO and decitabine in the treatment of thrombocytopenia following HSCT.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 97
• Est. completion date: April 12, 2019
• Est. primary completion date: April 12, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

1. Platelet count = 30 × 109/L persistently at day 60 post-HSCT or later;

2. Neutrophil and hemoglobin were well recovered;

3. Full donor chimerism was achieved;

4. No response to conventional treatments (e.g. thrombopoietin, immunoglobulin, glucocorticoid alone or in combination) for a duration of at least 4 weeks;

Exclusion Criteria:

1. Patients with malignancy relapse;

2. Active infections;

3. Grade ?-? acute GVHD or severe chronic GVHD according to National Institute of Health criteria;

4. Severe organ damage;

5. Thrombosis requiring treatment;

6. Received decitabine following the current transplantation.

Related Conditions & MeSH terms

• Hematologic Diseases
• Thrombocytopenia

Intervention

Drug:
• Decitabine
Decitabine
• rhTPO
rhTPO

Other:
• Conventional Treatment
immunoglobulin, glucocorticoid etc

Locations

Country	Name	City	State
China	The First affiliated Hospital of SooChow University	Suzhou	Jiangsu

Sponsors (9)

Lead Sponsor	Collaborator
The First Affiliated Hospital of Soochow University	Children's Hospital Of Soochow University, Fujian Medical University Union Hospital, Hebei Yanda Ludaopei Hospital, Nanfang Hospital of Southern Medical University, Peking University People's Hospital, Qilu Hospital of Shandong University, Ruijin Hospital, Wuhan Union Hospital, China

Country where clinical trial is conducted

China, 

References & Publications (3)

First LR, Smith BR, Lipton J, Nathan DG, Parkman R, Rappeport JM. Isolated thrombocytopenia after allogeneic bone marrow transplantation: existence of transient and chronic thrombocytopenic syndromes. Blood. 1985 Feb;65(2):368-74. — View Citation

Wang J, Yi Z, Wang S, Li Z. The effect of decitabine on megakaryocyte maturation and platelet release. Thromb Haemost. 2011 Aug;106(2):337-43. doi: 10.1160/TH10-11-0744. Epub 2011 Jun 28. — View Citation

Zhou H, Hou Y, Liu X, Qiu J, Feng Q, Wang Y, Zhang X, Min Y, Shao L, Liu X, Li G, Li L, Yang L, Xu S, Ni H, Peng J, Hou M. Low-dose decitabine promotes megakaryocyte maturation and platelet production in healthy controls and immune thrombocytopenia. Throm — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants With Platelet Count Recovery	Platelet response refers to a sustained increase (stable or increasing level) of at least 30×10E9/L independent of transfusion for 3 days.	Up to 4 weeks after the treatment
Secondary	Megakaryocyte Count	The total number of megakaryocytes as well as the platelet-shedding megakaryocytes of bone marrow smears (per cm2) was counted and cross-checked by blinded observers.	Up to 4 weeks after the treatment