Tacrolimus Treatment for Refractory PRCA

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

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

UNKNOWN

Clinical Phase

PHASE4

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2018-06-10

Study Completion Date

2020-06-10

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

Pure red cell aplasia (PRCA) is a kind of anemia characterized by severe reticulocytopenia and obvious bone marrow erythroblastic cells decreased. Cyclosporine and /or steroids are the first line therapy but some patients were refractory or intolerance to the treatment. The effects of the second line therapy are also not satisfactory and sometimes not available. The investigators aim to explore the efficacy and side-effect of tacrolimus for refractory PRCA.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Tacrolimus Treatment for Refractory Autoimmune Cytopenia

NCT03918265

Autoimmune Hemolytic Anemia Pure Red Cell Aplasia Evans Syndrome

UNKNOWN PHASE4

Assessment of Tolerance of Mobilizing Peripheral Hematopoietic Stem Cells by Plerixafor in Sickle Cell Patients

NCT02212535

Major Sickle Cell Syndrome of Type SS or Sβ Thalassemia

COMPLETED PHASE1/PHASE2

Sirolimus Combined With Low-dose Warfarin for the Treatment of Refractory PNH

NCT03866681

Paroxysmal Nocturnal Hemoglobinuria

UNKNOWN PHASE4

Study of MGTA-145 and Plerixafor in Patients With Sickle Cell Disease

NCT05445128

Sickle Cell Disease

TERMINATED PHASE2

Second-line Treatment of Primary Autoimmune Hemolytic Anemia

NCT05057468

Primary Autoimmune Hemolytic Anemia

RECRUITING PHASE3

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Pure red cell aplasia (PRCA) is a rare normocytic normochromic anemia with reticulocytopenia, characterized by a reduction of erythroid precursors from the bone marrow, could be divided into congenital and acquired PRCA according to pathogenesis. Congenital PRCA, also known as Diamond-Blackfan syndrome, has been associated with pathogenic variant in GATA1 and TSR2 and gene encode ribosomal proteins. Acquired PRCA can be a primary disease which is usually mediated by immunology, or secondary to other diseases, such as lymphoproliferative diseases, autoimmune diseases, thymoma, infection, or drugs. The first line therapy of acquired PRCA is Cyclosporine A and steroids, the second line therapy are anti-CD20, ATG, immunosuppressive drugs like cyclophosphamide, bone marrow transplantation. Unfortunately, some patients did not response or tolerate the above treatments. Tacrolimus, also known as FK506, is an agent mainly used after allogeneic organ transplant to lower risk of organ rejection. Tacrolimus could inhibit the production the production of IL-2, and also used in the therapy of other T cell mediated diseases. Tacrolimus primarily has been approved for prevent organ transplant rejection, especially in renal transplantation, tacrolimus also promises to treat autoimmune, degenerative and hyperproliferative disorders. Recently, tacrolimus has been reported to be effective and well tolerated for many immune-mediated cytopenias, such as autoimmune lymphoproliferative syndrome, immune thrombocytopenia, EVANS syndrome, etc. However, due to the rare occurrence of PRCA and good response rate to cyclosporine, there are very few studies of tacrolimus on refractory PRCA so far. In this study, it is anticipate to evaluate the effect of tacrolimus on 30 patients with refractory PRCA, the side-effects was documented and plasma concentration of tacrolimus will be monitor.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Pure Red Cell Aplasia

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

efficiency of tacrolimus on PRCA

A prospective research of the tacrolimus efficiency on refractory PRCA patients On refractory PRCA patients, tacrolimus was tried. Dosage: 1mg bid and tacrolimus trough targets were 5-10 ng/ml throughout the study.

Medication time should last at least 6 months.

Group Type EXPERIMENTAL

tacrolimus

Intervention Type DRUG

On refractory PRCA patients, tacrolimus was tried. Dosage: 1mg bid and tacrolimus trough targets were 5-10 ng/ml throughout the study

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

tacrolimus

On refractory PRCA patients, tacrolimus was tried. Dosage: 1mg bid and tacrolimus trough targets were 5-10 ng/ml throughout the study

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

tacrolimus capsule

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

Refractory pure red cell aplasia. 18-80 years old. No response or intolerant to first and second line therapies. Written informed consent.

Exclusion Criteria

Other diseases which might cause hematological abnormalities. Response and well tolerate to first or second line therapy. Patients who are under 18-year-old or over 80-year-old. Pregnant or lactating. Patients unwilling to or unable to comply with the protocol.

Minimum Eligible Age

18 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No