CARFILZOMIB, CYCLOPHOSPHAMIDE, DEXAMETHASONE: carfilzomib 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
Study summary
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.
Eligibility
Sex
ALL
Min age
18 Years
Max age
75 Years
Healthy volunteers
No
Inclusion Criteria:
1. Newly diagnosed POEMS syndrome meeting the Dispenzieri diagnostic criteria (2023 version);
2. Age 18-75 years;
3. ECOG performance status 0-3, with an estimated life expectancy \>3 months;
4. No active infective diseases;
5. No prior anti-POEMS therapy except for corticosteroids;
6. No severe organic impairment of major organs, meeting the following laboratory requirements: creatinine clearance ≥40 mL/min, total bilirubin ≤1.5 × upper limit of normal (ULN); AST and ALT ≤2.5 × ULN; cardiac enzymes \<2 × ULN; left ventricular ejection fraction within normal range on echocardiography, and no clinically significant electrocardiogram abnormalities;
7. Absolute neutrophil count ≥1.5 × 10\^9/L without prior growth factor support; platelet count ≥50 × 10\^9/L without platelet transfusion within 7 days prior to screening; hemoglobin ≥60 g/L;
8. Ability to swallow and take medication orally;
9. Completion of all screening and assessments as outlined in the study protocol;
10. Signed informed consent for chemotherapy.
Exclusion Criteria:
1. POEMS syndrome complicated by multiple myeloma, light chain amyloidosis, or Waldenström macroglobulinemia;
2. HIV positivity, or active hepatitis A, hepatitis B, or hepatitis C infection; or hepatitis B virus DNA \>10\^2 copies/mL;
3. 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;
4. 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;
5. Severe active infections (e.g., untreated tuberculosis, pulmonary aspergillosis);
6. Presence of other malignancies (except non-melanoma skin cancer, in situ cervical, bladder, or breast cancer with disease-free survival \>5 years);
7. Epilepsy requiring medication, dementia, or other mental status abnormalities that interfere with understanding or complying with the study protocol;
8. Drug use, medical, psychological, or social conditions that may interfere with study participation or outcome assessment;
9. Pregnancy or breastfeeding;
10. Any condition deemed by the investigator to make the patient unsuitable for enrollment.
Primary outcome measure(s)
Overall hematological response rate — 2 years 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 — 2 years 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 — 2 years 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
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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