Clinical trial NCT06254560: Rituximab for Serious Aplastic Anemia With Platelet Transfusion Refractoriness
Official title: The Study of Rituximab in the Treatment of Severe Aplastic Anemia With Platelet Transfusion Refractoriness
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Severe Aplastic Anemia
Platelet Transfusion Refractoriness
Keywords: Severe aplastic anemia
Platelet transfusion refractoriness
Rituximab
Lead sponsor: Institute of Hematology & Blood Diseases Hospital, China
Sponsor class: OTHER
First posted: 2024-02-12
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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

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

Primary outcomes:
measure: The response and complete remission rate with Rituximab protocol.
timeFrame: 6 months
Secondary outcomes:
measure: Relapse rate
timeFrame: 12 months and 60 months
measure: Sustained response (SR)
timeFrame: 12 months and 60 months
measure: Survival
timeFrame: 60 months
measure: Clonal evolution to myelodysplasia and acute leukemia.
timeFrame: 60 months
Other outcomes:
Locations:
city: Tianjin
country: China
facility: Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences
state: Tianjin
Study officials:
affiliation: Chinese Academy of Medical Sciences
name: Huang Jinbo, MD.
role: PRINCIPAL_INVESTIGATOR
References:
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.
citation: 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.
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.