Sirolimus Treatment for Newly Diagnosed Primary Acquired PRCA

NCT04470804 | Completed Phase 4

• 1 other identifier: PRCA-2
• Study Type: interventional
• Target: 56
• Locations: 1 country
• Sites: 1

Brief Summary

Pure red cell aplasia (PRCA) is a kind of anemia characterized by severe reticulocytopenia and obvious bone marrow erythroblastic cells decreased. Cyclosporine A 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 sirolimus for newly diagnosed primary acquired PRCA.

Conditions

Pure Red Cell Aplasia, Acquired

Keywords

sirolimus; newly diagnosed pure red cell aplasia; prospective study

Outcome Measures

Primary Outcomes (1)

• Hemoglobin level

  Hemoglobin level in g/L

  6 months

Secondary Outcomes (1)

• Hemoglobin level

  2 years

Other Outcomes (2)

• Overall response

  1 year

• Complete response

  1 year

Study Arms (2)

Sirolimus on newly diagnosed primary acquired PRCA

EXPERIMENTAL

A prospective research of the sirolimus efficiency on newly diagnosed primary acquired PRCA patients. Sirolimus dosage: 2mg QD with plasma concentration 4-15ng/mL. Medication time should last at least 6 months

Drug: Sirolimus

Cyclosporine A on newly diagnosed primary acquired PRCA

ACTIVE COMPARATOR

Cyclosporine A (CsA) efficiency on newly diagnosed primary acquired PRCA patients. CsA dosage: 4mg/kg QD. Medication time should last at least 6 months

Drug: Cyclosporine A

Interventions

Sirolimus DRUG

Sirolimus treats in experimental group

Also known as: Sirolimus Tablets

Sirolimus on newly diagnosed primary acquired PRCA

Cyclosporine A DRUG

Cyclosporine A uses for active comparator group.

Also known as: Cyclosporine A Tablets

Cyclosporine A on newly diagnosed primary acquired PRCA

Eligibility Criteria

• Age: 18 Years - 88 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• age \>18 years old.
• hemoglobin (Hb) \<90 g/L before treatment.
• no history of immunosuppression therapy prior to sirolimus or CsA treatment.
• adequate hepatic functions with alanine transaminase (ALT)/aspartate. transaminase (AST) levels within 3 times of the normal upper limit and total bilirubin levels within 2 times of the normal upper limit.
• documented patient consent.

You may not qualify if:

• diagnosis of secondary aPRCA.
• history of treatment with immunosuppression therapy before enrollment.
• history of leukemia, stem cell transplantation, or treatment-related myelodysplastic syndromes (MDS).
• creatinine/transaminase ≥ 3 normal upper limit.
• complicated with active or uncontrolled infections or uncontrolled cardiovascular disease.
• presence of other diseases that may cause anemia.
• presence of malignancies.
• pregnant and lactating women.

Sponsors & Collaborators

• Bing Han: lead

Study Sites (1)

Peking Union Medical College Hospital

Beijing, 100730, China

Location

Related Publications (7)

• Bride KL, Vincent T, Smith-Whitley K, Lambert MP, Bleesing JJ, Seif AE, Manno CS, Casper J, Grupp SA, Teachey DT. Sirolimus is effective in relapsed/refractory autoimmune cytopenias: results of a prospective multi-institutional trial. Blood. 2016 Jan 7;127(1):17-28. doi: 10.1182/blood-2015-07-657981. Epub 2015 Oct 26.

  PMID: 26504182 RESULT

• Li J, Wang Z, Dai L, Cao L, Su J, Zhu M, Yu Z, Bai X, Ruan C. Effects of rapamycin combined with low dose prednisone in patients with chronic immune thrombocytopenia. Clin Dev Immunol. 2013;2013:548085. doi: 10.1155/2013/548085. Epub 2013 Dec 2.

  PMID: 24363761 RESULT

• Means RT Jr. Pure red cell aplasia. Blood. 2016 Nov 24;128(21):2504-2509. doi: 10.1182/blood-2016-05-717140.

  PMID: 27881371 RESULT

• Teachey DT, Greiner R, Seif A, Attiyeh E, Bleesing J, Choi J, Manno C, Rappaport E, Schwabe D, Sheen C, Sullivan KE, Zhuang H, Wechsler DS, Grupp SA. Treatment with sirolimus results in complete responses in patients with autoimmune lymphoproliferative syndrome. Br J Haematol. 2009 Apr;145(1):101-6. doi: 10.1111/j.1365-2141.2009.07595.x. Epub 2009 Feb 4.

  PMID: 19208097 RESULT

• Long Z, Yu F, Du Y, Li H, Chen M, Zhuang J, Han B. Successful treatment of refractory/relapsed acquired pure red cell aplasia with sirolimus. Ann Hematol. 2018 Nov;97(11):2047-2054. doi: 10.1007/s00277-018-3431-5. Epub 2018 Jul 7.

  PMID: 29982851 RESULT

• Chen Z, Liu X, Chen M, Yang C, Han B. Successful sirolimus treatment of patients with pure red cell aplasia complicated with renal insufficiency. Ann Hematol. 2020 Apr;99(4):737-741. doi: 10.1007/s00277-020-03946-2. Epub 2020 Feb 6.

  PMID: 32030447 RESULT

• Yang Y, Tang Z, Huang Y, Hu Q, Wang S, Ji J, Du Y, Yang C, Chen M, Hu S, Han B. Sirolimus versus cyclosporine A in patients with primary acquired pure red cell aplasia: a prospective cohort study. Blood Cancer J. 2023 May 10;13(1):74. doi: 10.1038/s41408-023-00845-3. No abstract available.

  PMID: 37160872 DERIVED

MeSH Terms

Conditions

Red-Cell Aplasia, Pure

Interventions

Sirolimus; Cyclosporine

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals; Cyclosporins; Peptides, Cyclic; Macrocyclic Compounds; Polycyclic Compounds; Peptides; Amino Acids, Peptides, and Proteins

Study Officials

• Bing Han, PhD

  Peking Union Medical College Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: July 9, 2020
• First Posted: July 14, 2020
• Study Start: July 1, 2020
• Primary Completion: July 30, 2022
• Study Completion: July 31, 2022
• Last Updated: February 28, 2023

Record last verified: 2023-02

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)