About this trial
Platelet transfusion refractoriness (PTR) is a common complication in patients with hematological malignancies. It not only prolongs the duration of platelet transfusion dependence and significantly increases the risk of bleeding, but is also strongly associated with graft failure and reduced survival after transplantation. HLA class I antibody-mediated alloimmunization is recognized as the most important immunological cause of PTR. HLA antibodies are directly secreted by plasma cells, which are derived from B cells. Therefore, targeting B cells to reduce antibody production is a crucial step in eliminating HLA antibodies. Bruton's tyrosine kinase (BTK) is expressed throughout B cell development from the pre-B cell stage to maturity and supports B cell development, maturation, survival, proliferation, and antibody production by acting as a downstream kinase in the B cell receptor signaling pathway. Bortezomib, a proteasome inhibitor, can selectively induce apoptosis in long-lived plasma cells. The investigators' preliminary exploratory use of a BTK inhibitor in the treatment of PTR with HLA antibodies significantly reduced the mean fluorescence intensity (MFI) of HLA antibodies, improved platelet transfusion outcomes, and demonstrated a favorable safety profile. Based on these findings, the investigators are conducting a prospective, multicenter, randomized controlled two-arm study to investigate the efficacy and safety of acalabrutinib and bortezomib in eliminating HLA antibodies in hematological malignancies patients with PTR.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Patients with hematological malignancies and platelet transfusion refractoriness (24-hour CCI < 4.5×10⁹/L or PPR < 20%), and with the highest MFI of HLA antibodies > 8000 ;
Age 18-65 years, both male and female;
ECOG performance status 0-3;
Expected survival > 6 months;
Disqualifiers
Hypersensitivity to acalabrutinib, bortezomib, or excipients;
Major organ bleeding (central nervous system, lung, intestines) or grade ≥3 bleeding;
Hypersplenism;
Concurrent use of drugs that may cause excessive platelet consumption (amphotericin B, vancomycin, ATG, interferon, etc.);
Trial design
Parallel
Treatments tested in this trial
Acalabrutinib maleate
Drug100mg twice a day
bortezomib
Drug1.3mg/m2, d1,4,8,11
HLA-matched or crossmatched irradiated platelets
Other interventionTransfusion HLA-matched or crossmatched irradiated platelets 10U if platelet count lower than 10\*109/L
human immune globulin
Drug0.4g/kg.d for 5 days
Treatment groups
Trial outcomes
Primary outcomes
The response rate for anti-HLA antibody clearance
Complete response: HLA antibody MFI decrease ≥30% (applied to HLA antibody loci with baseline MFI \>8000; median value used for assessment) Partial response: HLA antibody MFI decrease ≥10% and \<30% No response: HLA antibody MFI decrease \<10%, or no decrease or even an increase.
CCI (corrected count increments)
CCI = (platelet increment per ul) x (body surface area in m2)/number of platelets transfused (x 10E11)
PPR (percentage platelet recovery)
PPR = Post-transfusion platelet count-pre-transfusion platelet count (/L) × total blood volume × 100%
Secondary outcomes
The incidence of bleeding events
The overall survival rate
Locations
Sponsors and contacts
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