Rituximab for Serious Aplastic Anemia With Platelet Transfusion Refractoriness

Severe Aplastic Anemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06254560
• Other study ID #: 2023NCRCA0113
• Status: Recruiting
• Phase: Phase 2
• Start date: February 23, 2023
• Est. completion date: December 30, 2027

Study information

• Verified date: February 2023
• Source: Institute of Hematology & Blood Diseases Hospital, China
• Contact: Huang Jinbo, MD.
• Phone: +86 22 23909023
• Email: huangjinbo[@]ihcams.ac.cn
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 20
• Est. completion date: December 30, 2027
• Est. primary completion date: December 30, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 70 Years

Eligibility

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

Related Conditions & MeSH terms

• Anemia
• Anemia, Aplastic
• Platelet Transfusion Refractoriness
• Severe Aplastic Anemia

Intervention

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.

Locations

Country	Name	City	State
China	Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences	Tianjin	Tianjin

Sponsors (1)

Lead Sponsor	Collaborator
Institute of Hematology & Blood Diseases Hospital, China

Country where clinical trial is conducted

China, 

References & Publications (3)

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. — View Citation

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. — View Citation

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. Erratum In: Br J Haematol. 2016 Nov;175(3):546. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Relapse rate	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.	12 months and 60 months
Secondary	Sustained response (SR)	SR was defined as Hb > 10 g/dL at 12 months and 60 months, in the absence of any treatment.	12 months and 60 months
Secondary	Survival	Survival rate within 5 years after diagnosis	60 months
Secondary	Clonal evolution to myelodysplasia and acute leukemia.	Clonal evolution within 5 years after diagnosis	60 months