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AZA Combined With NAC for PIT After HSCT

NCT ID: NCT05126004

Last Updated: 2021-11-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NOT_YET_RECRUITING

Clinical Phase

PHASE2

Total Enrollment

100 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-12-01

Study Completion Date

2026-06-30

Brief Summary

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Prolonged isolated thrombocytopenia (PIT) that is refractory to conventional treatments has remained a critical complication after allogeneic hematopoietic cell transplantation since decades years ago. Recombinant human thrombopoietin (rhTPO) is the main therapy in clinical practice, but remains low efficiency for PIT. Demethylating drugs have shown thier potential in high-risk myelodysplastic syndromes (MDS) and acte myeloid leukemia (AML). In addition, decitabine has demonstrated its efficacy of over 70% for response rate in treatment for PIT in early clinical trials with elusive mechanism. Preliminary experiments revealed that PIT was associated with abnormality of oxidation microenvironment, and N-Acetyl-L-cysteine (NAC) was the most commonly used antioxidant. Therefore, the investigators have been wondering whether Azacitidine in combination with NAC could improve PIT post HSCT and explore the possible mechanism of it.

Detailed Description

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Prolonged isolated thrombocytopenia (PIT) that is refractory to conventional treatments has remained a critical complication after allogeneic hematopoietic cell transplantation since decades years ago. Recombinant human thrombopoietin (rhTPO) is the main therapy in clinical practice, but remains low efficiency for PIT. Demethylating drugs have shown thier potential in high-risk myelodysplastic syndromes (MDS) and acte myeloid leukemia (AML). In addition, decitabine has demonstrated its efficacy of over 70% for response rate in treatment for PIT in early clinical trials with elusive mechanism. Preliminary experiments revealed that PIT was associated with abnormality of oxidation microenvironment, and N-Acetyl-L-cysteine (NAC) was the most commonly used antioxidant. Furthermore, AZA had shown its potential in immune regulation. Therefore, the investigators have been wondering whether Azacitidine in combination with NAC could improve PIT post HSCT and explore the possible mechanism of it.

Conditions

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Thrombocytopenia, Isolated Stem Cell Transplant Complications

Keywords

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Azacitidine N-Acetyl-L-cysteine Prolonged Isolated Thrombocytopenia Hematopoietic Stem Cell Transplantation

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Arm1: Control Arm2:AZA+NAC
Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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Control group

receive supportive care

Group Type NO_INTERVENTION

No interventions assigned to this group

AZA+NAC group

AZA 50mg Subcutaneous daily d1-d5 + NAC 600mg oral bid d1-28， 28 days for one cycle

Group Type EXPERIMENTAL

Azacitidine

Intervention Type DRUG

28 days for one cycle, evaluation post 3 cycles of treatment. Continue if response exists, otherwise, quit the trial and seek for other therapies.

N Acetyl L Cysteine

Intervention Type DRUG

28 days for one cycle, evaluation post 3 cycles of treatment. Continue if response exists, otherwise, quit the trial and seek for other therapies.

Interventions

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Azacitidine

28 days for one cycle, evaluation post 3 cycles of treatment. Continue if response exists, otherwise, quit the trial and seek for other therapies.

Intervention Type DRUG

N Acetyl L Cysteine

28 days for one cycle, evaluation post 3 cycles of treatment. Continue if response exists, otherwise, quit the trial and seek for other therapies.

Intervention Type DRUG

Other Intervention Names

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AZA NAC

Eligibility Criteria

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Inclusion Criteria

* Platelet count ≤ 30 × 10\^9/L persistently at day 60 post-HSCT or later;
* Neutrophil and hemoglobin were well recovered;
* Full donor chimerism was achieved;

Exclusion Criteria

* Patients with malignancy relapse;
* Active infections;
* Grade Ⅲ-Ⅳ acute graft-versus-host disease or severe chronic graft-versus-host disease according to National Institute of Health criteria;
* Severe organ damage;
* Thrombosis requiring treatment;
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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The First Affiliated Hospital of Soochow University

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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The first affiliated hospital of Soochow University

Suzhou, Jiangsu, China

Site Status

Countries

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China

Central Contacts

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Yue Han, PhD,MD

Role: CONTACT

Phone: 0512-67781856

Email: [email protected]

Depei Wu, PhD,MD

Role: CONTACT

Facility Contacts

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Yue Han, PhD, MD

Role: primary

Meng Zhou, MD

Role: backup

References

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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.

Reference Type BACKGROUND
PMID: 3881142 (View on PubMed)

Bruno B, Gooley T, Sullivan KM, Davis C, Bensinger WI, Storb R, Nash RA. Secondary failure of platelet recovery after hematopoietic stem cell transplantation. Biol Blood Marrow Transplant. 2001;7(3):154-62. doi: 10.1053/bbmt.2001.v7.pm11302549.

Reference Type BACKGROUND
PMID: 11302549 (View on PubMed)

Wang H, Huang M, Zhao Y, Qi JQ, Chen C, Tang YQ, Qiu HY, Fu CC, Tang XW, Wu DP, Ruan CG, Han Y. Recombinant Human Thrombopoietin Promotes Platelet Engraftment and Improves Prognosis of Patients with Myelodysplastic Syndromes and Aplastic Anemia after Allogeneic Hematopoietic Stem Cell Transplantation. Biol Blood Marrow Transplant. 2017 Oct;23(10):1678-1684. doi: 10.1016/j.bbmt.2017.06.010. Epub 2017 Jun 19.

Reference Type BACKGROUND
PMID: 28642072 (View on PubMed)

Zhao X, Zhao X, Huo M, Fan Q, Pei X, Wang Y, Zhang X, Xu L, Huang X, Liu K, Chang Y. Donor-Specific Anti-Human Leukocyte Antigen Antibodies Predict Prolonged Isolated Thrombocytopenia and Inferior Outcomes of Haploidentical Hematopoietic Stem Cell Transplantation. J Immunol Res. 2017;2017:1043836. doi: 10.1155/2017/1043836. Epub 2017 Apr 16.

Reference Type BACKGROUND
PMID: 28484721 (View on PubMed)

Ramirez P, Brunstein CG, Miller B, Defor T, Weisdorf D. Delayed platelet recovery after allogeneic transplantation: a predictor of increased treatment-related mortality and poorer survival. Bone Marrow Transplant. 2011 Jul;46(7):981-6. doi: 10.1038/bmt.2010.218. Epub 2010 Oct 4.

Reference Type BACKGROUND
PMID: 20921943 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 33646303 (View on PubMed)

Mohyeldin A, Garzon-Muvdi T, Quinones-Hinojosa A. Oxygen in stem cell biology: a critical component of the stem cell niche. Cell Stem Cell. 2010 Aug 6;7(2):150-61. doi: 10.1016/j.stem.2010.07.007.

Reference Type BACKGROUND
PMID: 20682444 (View on PubMed)

Chen S, Su Y, Wang J. ROS-mediated platelet generation: a microenvironment-dependent manner for megakaryocyte proliferation, differentiation, and maturation. Cell Death Dis. 2013 Jul 11;4(7):e722. doi: 10.1038/cddis.2013.253.

Reference Type BACKGROUND
PMID: 23846224 (View on PubMed)

Kong Y, Shi MM, Zhang YY, Cao XN, Wang Y, Zhang XH, Xu LP, Huang XJ. N-acetyl-L-cysteine improves bone marrow endothelial progenitor cells in prolonged isolated thrombocytopenia patients post allogeneic hematopoietic stem cell transplantation. Am J Hematol. 2018 Jul;93(7):931-942. doi: 10.1002/ajh.25056. Epub 2018 Feb 24.

Reference Type BACKGROUND
PMID: 29396859 (View on PubMed)

Han Y, Tang Y, Chen J, Liang J, Ye C, Ruan C, Wu D. Low-Dose Decitabine for Patients With Thrombocytopenia Following Allogeneic Hematopoietic Stem Cell Transplantation: A Pilot Therapeutic Study. JAMA Oncol. 2015 May;1(2):249-51. doi: 10.1001/jamaoncol.2014.316. No abstract available.

Reference Type BACKGROUND
PMID: 26181032 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 25566808 (View on PubMed)

Provided Documents

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Document Type: Informed Consent Form

View Document

Other Identifiers

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SOOCHOW-HY-2021-2

Identifier Type: -

Identifier Source: org_study_id