Prospective Study of Patients With Thrombocytopenia Following HSCT

NCT02487563 | Completed Phase 3 Results DMC

• 1 other identifier: SOOCHOW-HY-2015
• Study Type: interventional
• Target: 97
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Thrombocytopenia; Hematologic Diseases

Keywords

Isolated thrombocytopenia; decitabine; rhTPO

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Megakaryocyte Count

  Up to 4 weeks after the treatment

Study Arms (3)

Experimental Group 1

EXPERIMENTAL

Decitabine in combination with rhTPO.

Drug: Decitabine; Drug: rhTPO

Experimental Group 2

EXPERIMENTAL

Decitabine

Drug: Decitabine

Control Group

ACTIVE COMPARATOR

Conventional treatment except decitabine.

Other: Conventional Treatment

Interventions

Decitabine DRUG

Decitabine

Also known as: 5-Aza-2'-deoxycytidine

Experimental Group 1; Experimental Group 2

rhTPO DRUG

rhTPO

Also known as: Thrombopoietin

Experimental Group 1

Conventional Treatment OTHER

immunoglobulin, glucocorticoid etc

Control Group

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Platelet count ≤ 30 × 109/L persistently at day 60 post-HSCT or later;
• Neutrophil and hemoglobin were well recovered;
• Full donor chimerism was achieved;
• No response to conventional treatments (e.g. thrombopoietin, immunoglobulin, glucocorticoid alone or in combination) for a duration of at least 4 weeks;

You may not qualify if:

• Patients with malignancy relapse;
• Active infections;
• Grade Ⅲ-Ⅳ acute GVHD or severe chronic GVHD according to National Institute of Health criteria;
• Severe organ damage;
• Thrombosis requiring treatment;
• Received decitabine following the current transplantation.

Sponsors & Collaborators

• The First Affiliated Hospital of Soochow University: lead
• Peking University People's Hospital: collaborator
• Nanfang Hospital, Southern Medical University: collaborator
• Ruijin Hospital: collaborator
• Union Hospital, Tongji Medical College, Huazhong University of Science and Technology: collaborator
• Qilu Hospital of Shandong University: collaborator
• Children's Hospital of Soochow University: collaborator
• Fujian Medical University Union Hospital: collaborator
• Hebei Yanda Ludaopei Hospital: collaborator

Study Sites (1)

The First affiliated Hospital of SooChow University

Suzhou, Jiangsu, 215000, China

Location

Related Publications (4)

• 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.

  PMID: 3881142 RESULT

• 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.

  PMID: 21713321 RESULT

• 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. Thromb Haemost. 2015 May;113(5):1021-34. doi: 10.1160/TH14-04-0342. Epub 2015 Jan 8.

  PMID: 25566808 RESULT

• Tang Y, Chen J, Liu Q, Chu T, Pan T, Liang J, He XF, Chen F, Yang T, Ma X, Wu X, Hu S, Cao X, Hu X, Hu J, Liu Y, Qi J, Shen Y, Ruan C, Han Y, Wu D. Low-dose decitabine for refractory prolonged isolated thrombocytopenia after HCT: a randomized multicenter trial. Blood Adv. 2021 Mar 9;5(5):1250-1258. doi: 10.1182/bloodadvances.2020002790.

  PMID: 33646303 DERIVED

MeSH Terms

Conditions

Thrombocytopenia; Hematologic Diseases

Interventions

Decitabine; Thrombopoietin

Condition Hierarchy (Ancestors)

Blood Platelet Disorders; Hemic and Lymphatic Diseases; Cytopenia

Intervention Hierarchy (Ancestors)

Azacitidine; Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides; Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Limitations and Caveats

Small number of patients, the diversity of patients, and the heterogeneous treatment options in Arm C.

Results Point of Contact

• Title: Yaqiong Tang
• Organization: Jiangsu Institute of Hematology, The First Affiliated Hospital of Soochow University

tangyaqiong@suda.edu.cn

+86 18896588075

Study Officials

• Depei Wu, PhD,MD

  The First Affiliated Hospital of Soochow University

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 21, 2015
• First Posted: July 1, 2015
• Study Start: October 1, 2015
• Primary Completion: April 12, 2019
• Study Completion: April 12, 2019
• Last Updated: May 6, 2020
• Results First Posted: May 6, 2020

Record last verified: 2020-04

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)