Rituximab for Serious Aplastic Anemia With Platelet Transfusion Refractoriness

NCT06254560 | Recruiting Phase 2

• 1 other identifier: 2023NCRCA0113
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

Due to long-term dependence on platelet transfusion, some severe aplastic anemia (SAA) patients suffer platelet transfusion refractoriness (PTR). Unlike immune thrombocytopenia (ITP), glucocorticoids and human immunoglobulin (IVIg) are generally ineffective for PTR. Due to the lack of effective intervention methods, patients with PTR suffer increased platelet transfusions, bleeding events and treatment costs, prolonged hospital stays, and decreased survival rate. SAA with PTR has become a challenge for physicians. The experiment aims to explore the efficacy of rituximab in the treatment of SAA with PTR, and establish a new effective, safe treatment method with relatively low treatment cost.

Conditions

Severe Aplastic Anemia; Platelet Transfusion Refractoriness

Keywords

Severe aplastic anemia; Platelet transfusion refractoriness; Rituximab

Outcome Measures

Primary Outcomes (1)

• The response and complete remission rate with Rituximab protocol.

  Response will be evaluated at each clinic visit. Complete response (CR) was defined as achieving all three peripheral blood count criteria: (1) Hb level up to the normal range; (2) ANC≥1.5×109/L; (3) PLT≥100×109/L. Partial response (PR) was defined as transfusion independent, no longer meeting criteria for severe disease. Persistence of transfusion requirement or death was evidence of no response (NR).

  6 months

Secondary Outcomes (4)

• Relapse rate

  12 months and 60 months

• Sustained response (SR)

  12 months and 60 months

• Survival

  60 months

• Clonal evolution to myelodysplasia and acute leukemia.

  60 months

Study Arms (1)

rituximab group

EXPERIMENTAL

Rituximab combined with cyclosporine

Drug: Rituximab

Interventions

Rituximab DRUG

Rituximab is administered at a dose of 100mg per week, a total of 4 times. Cyclosporin is administered at a dose of 3-5mg/kg per day.

Also known as: Mabthera

rituximab group

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Initial diagnosed SAA with PTR
• Age\>18 years old, regardless of gender
• Initial diagnosed SAA with PTR
• Age\>18 years old, regardless of gender

You may not qualify if:

• Allergy to rituximab
• Severe active infection
• Hypogammaglobulinemia
• Pregnant and lactating women
• Heart failure (NYHA classification IV)
• Individuals with epilepsy, dementia, and other mental disorders that require medication treatment who cannot understand or follow the research protocol
• Chronic infections or other chronic diseases that may be risk to the experiment
• The researchers believe that it is not suitable for participants

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

Study Sites (1)

Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences

Tianjin, Tianjin Municipality, 300020, China

RECRUITING

Related Publications (3)

• Killick SB, Bown N, Cavenagh J, Dokal I, Foukaneli T, Hill A, Hillmen P, Ireland R, Kulasekararaj A, Mufti G, Snowden JA, Samarasinghe S, Wood A, Marsh JC; British Society for Standards in Haematology. Guidelines for the diagnosis and management of adult aplastic anaemia. Br J Haematol. 2016 Jan;172(2):187-207. doi: 10.1111/bjh.13853. Epub 2015 Nov 16. No abstract available.

  PMID: 26568159 BACKGROUND

• Chockalingam P, Sacher RA. Management of patients refractory to platelet transfusion. J Infus Nurs. 2007 Jul-Aug;30(4):220-5. doi: 10.1097/01.NAN.0000281531.97183.c0.

  PMID: 17667077 BACKGROUND

• Kerkhoffs JL, Eikenboom JC, van de Watering LM, van Wordragen-Vlaswinkel RJ, Wijermans PW, Brand A. The clinical impact of platelet refractoriness: correlation with bleeding and survival. Transfusion. 2008 Sep;48(9):1959-65. doi: 10.1111/j.1537-2995.2008.01799.x. Epub 2008 Jun 28.

  PMID: 18564396 BACKGROUND

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

Rituximab

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Murine-Derived; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Officials

• Huang Jinbo, MD.

  Chinese Academy of Medical Sciences

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Huang Jinbo, MD.

CONTACT

+86 22 23909023; huangjinbo@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 19, 2024
• First Posted: February 12, 2024
• Study Start: February 23, 2023
• Primary Completion (Estimated): December 30, 2026
• Study Completion (Estimated): December 30, 2027
• Last Updated: February 12, 2024

Record last verified: 2023-02

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)