This study is a single-center, prospective, open-label clinical study to evaluate the efficacy and safety of KCD(Carfilzomib/Cyclophosphamide/Dexamethasone) regimen in subjects with newly diagnosed POEMS Syndrome.
Newly diagnosed POEMS syndrome meeting the Dispenzieri diagnostic criteria (2023 version);
Age 18-75 years;
ECOG performance status 0-3, with an estimated life expectancy >3 months;
No active infective diseases;
POEMS syndrome complicated by multiple myeloma, light chain amyloidosis, or Waldenström macroglobulinemia;
HIV positivity, or active hepatitis A, hepatitis B, or hepatitis C infection; or hepatitis B virus DNA >10² copies/mL;
Concurrent severe unstable medical conditions, including heart failure, renal failure, liver failure, bleeding disorders, arterial/venous thrombotic events within 6 months, uncontrolled diabetes mellitus, or a history of active hemorrhagic cystitis;
History of autoimmune diseases (e.g., Crohn's disease, rheumatoid arthritis, systemic lupus erythematosus) within the past 2 years that caused end-organ damage or required systemic immunosuppressive or disease-modifying therapy;
Single group
CARFILZOMIB, CYCLOPHOSPHAMIDE, DEXAMETHASONE
Drugcarfilzomib at a dose of 27 mg/m2 (20 mg/m2 only in the first infusion) intravenously (iv) on days 1, 8, and 15,cyclophosphamide at a dose of 200 mg/m2 iv on days 1, 8 and 15 and dexamethasone at a dose of 20 mg (10 mg for patients \>75 years) days 1, 2, 8, 9, 15 and 16
Overall hematological response rate
hematological response rate: * Complete Remission (CR\_H): Normal bone marrow; negative serum and urine immunofixation electrophoresis; disappearance of M-protein. * Very Good Partial Remission (VGPR\_H): Reduction of M-protein by \>90% (baseline M-protein ≥5 g/L). * Partial Remission (PR\_H): Reduction of M-protein by ≥50% (baseline M-protein ≥10 g/L). * No Response (NR\_H): Failure to meet criteria for PR\_H. * Progressive Disease (PD): Reappearance of M-protein in serum and/or urine, or an increase of \>25% from the lowest level (with absolute M-protein increase ≥5 g/L).
Overall VEGF response rate
VEGF response rate: * Complete Remission (CR\_V): Normalization of serum VEGF (elevation typically defined as serum VEGF \>2 times the upper limit of normal). * Partial Remission (PR\_V): Reduction of VEGF by ≥50%. * No Response (NR\_V): Failure to meet criteria for PR\_V. * Progressive Disease (PD): Persistent (≥2 consecutive measurements) elevation of VEGF , or persistent elevation of VEGF by 50% from the post-treatment nadir.
Overall neurological response rate
Neurological response rate: Neurologic improvement assessed by neurophysiologic examination, modified Rankin Scale, or Overall Neuropathy Limitations Scale (ONLS). 1. Complete response: 0 point; 2. Improvement: Improved by 1 point; 3. Progression: Worsened by 1 point
the incidence of adverse events and severe adverse events
Major Adverse Events 1. Hematologic toxicity: neutropenia, thrombocytopenia, etc. 2. Infections: bacterial pneumonia, sepsis, etc. 3. Worsening of neuropathy 4. Capillary leak syndrome 5. Thrombotic events Grading and Definition of Serious Adverse Event All adverse events are graded according to the Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0. Gr
the 2-year overall survival rate
Two-year overall survival (2-year OS) is defined as the proportion of patients who remain alive at two years following the start of treatment (or from the date of diagnosis).
Two-year progression-free survival
Two-year progression-free survival (2-year PFS) is defined as the proportion of patients who remain alive and have not experienced disease progression at two years following the start of treatment (or from the time of diagnosis)
Click on the lead sponsor to view all of their trials.
