A Multicenter, Prospective, Randomized Controlled Study Comparing Glucocorticoid Combined With Sirolimus With Monotherapy of Glucocorticoid in the Treatment of Newly Diagnosed Mild Autoimmune Hemolytic Anemia

NCT07518277 | Not Yet Recruiting

• 1 other identifier: GC vs. GC+Sirolimus
• Study Type: interventional
• Target: 216
• Locations: 1 country
• Sites: 1

Brief Summary

This study is a prospective, multicenter, randomized controlled trial. A total of 216 adult patients with newly diagnosed wAIHA were planned to be included and randomly assigned in a 1:1 ratio to the experimental group (glucocorticoid combined with sirolimus) or the control group (glucocorticoid monotherapy). The initial dose of sirolimus in the experimental group was 1mg/d, adjusted according to the blood drug concentration. The target concentration was 4-12ng/mL, and the treatment course was 6 months. Both groups of hormones were gradually reduced according to the standard protocol. All patients were followed up for 24 months, and the differences between the two groups at endpoints such as the hormone-free sustained response rate at the 12th month were compared.

Conditions

Autoimmune Hemolytic Anemia (AIHA)

Keywords

autoimmune hemolytic anemia

Outcome Measures

Primary Outcomes (1)

• The 12-month sustained remission rate

  Defined as the proportion of patients who simultaneously met the following conditions at the 12th month after randomization: Discontinue glucocorticoids (or only use prednisone ≤5 mg/d or an equivalent dose for maintenance); * Has not received any follow-up treatment for wAIHA; * Still in complete remission (CR) or partial remission (PR) status.

  12 months

Secondary Outcomes (1)

• Safety

  1，3，6，12 months

Study Arms (2)

Glucocorticoid

ACTIVE COMPARATOR

Glucocorticoid monotherapy group Prednisone at a dose of 1-2 mg/kg/d (or an equivalent dose of methylprednisolone) is taken orally for 2-3 weeks, and then the dosage is reduced regularly. The total course of treatment is approximately 3-6 months. The reduction plan was formulated by the researchers based on clinical practice.

Drug: Glucocorticoids

Glucocorticoids combined with Sirolimus

EXPERIMENTAL

Glucocorticoids: The usage, dosage and reduction regimen are the same as those in the monotherapy group. \| Sirolimus: Starting dose 1 mg/d, orally, once a day. After reaching a steady state 7 to 14 days of medication, monitor the trough concentration. The target trough concentration range is 4 to 12 ng/mL. Adjust the dosage based on the results of blood drug concentration monitoring. The total course of treatment for sirolimus is 6 months. If grade ≥3 related adverse events or trough concentration \&gt occur; 12 ng/mL. Consider reducing the dosage or suspending the administration.

Drug: Glucocorticoids

Interventions

Glucocorticoids DRUG

Glucocorticoids combined with sirolimus

Glucocorticoid; Glucocorticoids combined with Sirolimus

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥18 years old, gender not limited;
• Diagnosed as newly diagnosed wAIHA and requiring systemic immunosuppressive therapy (those with short-term hormone exposure of ≤2 weeks before enrollment can be included)
• After a comprehensive rheumatology and immunology assessment, there is no evidence of clinical organ involvement in SLE or other connective tissue diseases (only serological abnormalities can be included).
• Positive Coombs test (IgG type, IgG+C3d type, or only C3d type with a condensed agglutinin titer of 1:64);
• Active hemolytic anemia, hemoglobin (HGB) ≤ 100 g/L;
• Liver and kidney function: Alanine aminotransferase (ALT) \< 3 times the upper limit of the normal value (ULN), isolated elevation of aspartate aminotransferase (AST) (normal ALT) is acceptable; Serum creatinine ≤2×ULN;
• Eastern Cooperative Oncology Group (ECOG) Performance status score ≤2 points;
• Those with a history of malignant tumors must meet the following conditions:
• Radical treatment has been achieved; The disease-free survival period complies with the protocol provisions (such as ≥1 year for basal cell carcinoma, ≥5 years for early solid tumors, ≥5 years for curable lymphoma, etc.); There is currently no evidence of recurrence. Those who do not meet the above conditions (such as CLL, advanced solid tumors, and active tumors) are not included.
• Voluntarily sign a written informed consent form.

You may not qualify if:

• Pregnant or lactating patients;
• Clinically confirmed SLE or other definite connective tissue diseases;
• Secondary AIHA secondary to lymphoproliferative diseases, other hematological malignancies, solid tumors, infections, and drugs;
• Cold-resistant type or hybrid type AIHA;
• Received before group enrollment:
• Other immunosuppressants (such as rituximab, cyclosporine, etc., regardless of the duration); Glucocorticoid treatment for more than 2 weeks;
• Severe cardiac insufficiency (NYHA grade III/IV, or LVEF \< 40%);
• Chronic active infections (active tuberculosis, active hepatitis B/C, etc.);
• Severe immunodeficiency diseases, HIV infection (CD4⁺ \< 200/μL), currently using other potent immunosuppressants, and using targeted biological agents within the last 3 months;
• Have a history of severe allergy to sirolimus, glucocorticoids or related excipients;
• Other circumstances where the researcher deems it inappropriate to participate.

Sponsors & Collaborators

• Chen Miao: lead

Study Sites (1)

Peking union medical college hospital

Beijing, Shuangfuyuan, NO I., 100730, China

Location

Related Publications (1)

• 1.Birgens H, Frederiksen H, Hasselbalch HC, et al. A phase III randomized trial comparing glucocorticoid monotherapy versus glucocorticoid and rituximab in patients with autoimmune haemolytic anaemia. Br J Haematol. 2013;163(2):244-252.https://doi.org/10.1111/bjh.12541. 2.Kuter DJ. Warm autoimmune hemolytic anemia and the best treatment strategies. Hematology Am Soc Hematol Educ Program. 2022;2022(1):393-402.https://doi.org/10.1182/hematology.2022000405. 3.Murakhovskaya I, Crivera C, Leon A,et al. Healthcare resource utilization of patients with warm autoimmune hemolytic anemia initiating first line therapy of oral corticosteroids with or without rituximab. Ann Hematol. 2024;103(2):521-533.https://doi.org/10.1007/s00277-023-05613-8. 4.Berentsen S. Treatment of autoimmune hemolytic anemia: novel and investigational approaches. Minerva Med. 2025;116(2):145-158.https://doi.org/10.23736/S0026-4806.25.09617-X. 5.Ciudad M, Ouandji S, Lamarthée B, et al. Regulatory T-cell dysfunctions are associated with increase in tumor necrosis factor α in autoimmune hemolytic anemia and participate in polarization. Haematologica. 2023;108(11):3012-3025. https://doi.org/10.3324/haematol.2023.282859. 6.中华医学会血液学分会红细胞疾病（贫血）学组. 中国成人自身免疫性溶血性贫血诊疗指南（2023年版）[J]. 中华血液学杂志,2023,44(01)：12-18. DOI:10.3760/cma.j.issn.0253-2727.2023.01.003

  BACKGROUND

MeSH Terms

Conditions

Anemia, Hemolytic, Autoimmune

Interventions

Glucocorticoids

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Adrenal Cortex Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Physiological Effects of Drugs; Pharmacologic Actions; Chemical Actions and Uses

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Peking Union Medical College Hospital

Study Record Dates

• First Submitted: March 30, 2026
• First Posted: April 8, 2026
• Study Start: April 1, 2026
• Primary Completion (Estimated): April 1, 2028
• Study Completion (Estimated): April 1, 2028
• Last Updated: April 8, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)