Tacrolimus Treatment for Refractory Autoimmune Cytopenia

Chinese Academy of Medical Sciences & Peking Union Medical College

Status and phase: Unknown; Phase 4

Conditions: Evans Syndrome; Pure Red Cell Aplasia; Autoimmune Hemolytic Anemia

Treatments: Drug: Tacrolimus

Study type: Interventional

Funder types: Other

Identifiers

NCT03918265

tacrolimus-2

Details and patient eligibility

About

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.

Full 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.

Enrollment: 80 estimated patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No 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.

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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80 participants in 1 patient group

Efficiency of tacrolimus on autoimmune cytopenia

Experimental group

Description:

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.

Treatment:

Drug: Tacrolimus