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NCT Number: NCT06254560

Rituximab for Serious Aplastic Anemia With Platelet Transfusion Refractoriness

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.

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Key information

Conditions

Severe Aplastic Anemia Anemia Anemia, Aplastic Bone Marrow Diseases Bone Marrow Failure Disorders Hematologic Diseases Hemic and Lymphatic Diseases Platelet Transfusion Refractoriness

Age range

18 year–70 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

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

Tianjin, Tianjin Municipality, 300020, China

Location status: Recruiting

Location contact

Huang Jinbo, M.D.

CONTACT

[email protected]

+86 22 23909023

About this study

During the treatment period, Rituximab is administered at a dose of 100mg per week, a total of 4 times.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

  • 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

Treatment and study plan

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.

Other names: Mabthera

Primary outcomes

  1. The response and complete remission rate with Rituximab protocol.

    Time frame: 6 months

    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).

Secondary outcomes

  1. Relapse rate

    Time frame: 12 months and 60 months

    Relapse was defined as a responder who met criteria for SAA again after achieving response and keeping stable blood counts for at least 3 months.

  2. Sustained response (SR)

    Time frame: 12 months and 60 months

    SR was defined as Hb > 10 g/dL at 12 months and 60 months, in the absence of any treatment.

  3. Survival

    Time frame: 60 months

    Survival rate within 5 years after diagnosis

  4. Clonal evolution to myelodysplasia and acute leukemia.

    Time frame: 60 months

    Clonal evolution within 5 years after diagnosis

Study contacts

Contact information is provided by the study sponsor or research team.

Huang Jinbo, MD.

CONTACT

[email protected]

+86 22 23909023

Sponsors and collaborators

Lead sponsor

Institute of Hematology & Blood Diseases Hospital, China

Other

Registry information

Official study title

The Study of Rituximab in the Treatment of Severe Aplastic Anemia With Platelet Transfusion Refractoriness

Important dates

Study start
2023
Primary completion
2026
Study completion
2027
First posted
Feb 12, 2024
Registry last updated
Feb 12, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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