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Recruiting NCT06254560

Rituximab for Serious Aplastic Anemia With Platelet Transfusion Refractoriness

Phase II Interventional Severe Aplastic Anemia Platelet Transfusion Refractoriness

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Rituximab.
Who it may be relevant to
Registry conditions: Severe Aplastic Anemia, Platelet Transfusion Refractoriness. Basic parameters: 18 years — 70 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
China
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Official title

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

Overview

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.

Detailed description

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

Interventions

  • Drug Rituximab
    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.

Primary outcome measures

  • The response and complete remission rate with Rituximab protocol. [Time frame: 6 months]
Secondary outcome measures (4)
  • Relapse rate [Time frame: 12 months and 60 months]
  • Sustained response (SR) [Time frame: 12 months and 60 months]
  • Survival [Time frame: 60 months]
  • Clonal evolution to myelodysplasia and acute leukemia. [Time frame: 60 months]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

China · 1 center
  • Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences — Tianjin

Publications

  • 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
  • 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
  • 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

Identifiers

NCT: NCT06254560 · 2023NCRCA0113

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗