Tacrolimus Treatment for Refractory Autoimmune Cytopenia

Sponsor

Peking Union Medical College Hospital (Other)

Overall Status

Unknown status

CT.gov ID

NCT03918265

Collaborator

(none)

Enrollment

Location

Arm

30.3

Anticipated Duration (Months)

2.6

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Autoimmune cytopenia, including autoimmune hemolytic anemia (AIHA), pure red cell aplasia (PRCA), Evans syndrome (ES), usually has good responses to steroids therapies as first line, but there is a considerable percentage of patients who relapse, become refractory or dependent on steroids to maintain an acceptable level of hemoglobin or platelets. 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 autoimmune cytopenia.

Condition or Disease	Intervention/Treatment	Phase
Autoimmune Hemolytic Anemia Pure Red Cell Aplasia Evans Syndrome	Drug: Tacrolimus	Phase 4

Detailed Description

Tacrolimus binds FKBP12 with high affinity after entry into cytoplasm, suppresses calcineurin activity and prevents nuclear translocation of transcription factors such as NF-AT that are involved in IL-2 gene transcription. As a result, T-cell activation is inhibited with a subsequent reduction in the production of cytokines that include IL-2, TNF-α, IL-3, IL-4, IFN-γ, IL-6 and IL-10; B-cell activation, class-switching and immunoglobulin production are also attenuated. Reports on the use of tarcrolimus in the treatment of autoimmune cytopenia are anecdotal and describe therapy with a variety of drug dosages.

The purpose of this study is to evaluate the effect of tacrolimus on patients with refractory autoimmune cytopenia, the side-effects will be documented and plasma concentration of tacrolimus will be monitor.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

80 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Tacrolimus Treatment for Refractory Autoimmune Cytopenia

Actual Study Start Date :

May 4, 2019

Anticipated Primary Completion Date :

Aug 10, 2020

Anticipated Study Completion Date :

Nov 10, 2021

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Efficiency of tacrolimus on autoimmune cytopenia A prospective research of the tacrolimus efficiency on refractory autoimmune cytopenia patients. Dosage: 1mg bid and tacrolimus trough targets were 5-10 ng/ml throughout the study. Medication time should last at least 6 months.	Drug: Tacrolimus On refractory autoimmune cytopenia patients, tacrolimus was tried. Dosage: 1mg bid and tacrolimus trough targets were 5-10 ng/ml throughout the study Other Names: tacrolimus capsule

Arm

Intervention/Treatment

Experimental: Efficiency of tacrolimus on autoimmune cytopenia

A prospective research of the tacrolimus efficiency on refractory autoimmune cytopenia patients. Dosage: 1mg bid and tacrolimus trough targets were 5-10 ng/ml throughout the study. Medication time should last at least 6 months.

Drug: Tacrolimus

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

Other Names:

tacrolimus capsule

Outcome Measures

Primary Outcome Measures

Hemoglobin level [6 months]
Hemoglobin level in g/L
Platelet count [6 months]
Platelet count in /L

Secondary Outcome Measures

Hemoglobin level [2 years]
Hemoglobin level in g/L
Platelet count [2 years]
Platelet count in /L

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 80 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Refractory autoimmune cytopenia, including autoimmune hemolytic anemia, pure red cell aplasia, Evans syndrome.
18-80 years old.
No response or intolerant to first and second line therapies.
ECOG Performance Status of 0-2
Written informed consent.