Rapid dFLC Response Predict Complete Hematologica Response in Systemic AL Amyloidosis Patients Treated With Daratumumab-based Regimen
Overview
- Phase
- Not Applicable
- Status
- Recruiting
- Enrollment
- 50
- Locations
- 8
- Primary Endpoint
- Complete hematologic response
Overview
Brief Summary
Light chain amyloidosis (AL amyloidosis) is a rare plasma cell dyscrasia characterized by the deposition of insoluble amyloid fibrils in multiple organ systems. The treatment of amyloidosis primarily relies on anti-plasma cell therapy and supportive care. The application of anti-plasma cell therapy has significantly improved outcomes in patients with AL amyloidosis. Standard first-line therapy typically includes daratumumab, bortezomib, cyclophosphamide, and dexamethasone (Dara-BCD), achieving a complete hematologic response in nearly 60% of patients.The depth and speed of the hematologic response are strongly correlated with organ response and overall survival. An early achievement of a complete hematologic response is particularly crucial in cases of AL amyloidosis characterized by significant organ involvement. The median time to a hematologic response for the daratumumab based treatment is only 7-9 days. The retrospective data showed that the hematologic response in Day 7 in Cycle 1 (C1D7) may predict the complete hematologic response rate. In order to validate the conclusion, the investigator design this prospective study.
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 18 Years to 99 Years (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Diagnosis of systemic AL amyloidosis;
- •Daratumumab, bortezomib, dexamethasone used in treatment;
- •Informed consent explained to, understood by and signed by the patient;
- •dFLC ≥ 50 mg/L;
Exclusion Criteria
- •Fulfill with the criteria of active multiple myeloma or active lymphoplasmacytic lymphoma;
- •Presence of other tumors which is/are in advanced malignant stage and has/have systemic metastasis;
- •Severe or persistent infection that cannot be effectively controlled;
- •Presence of severe autoimmune diseases or immunodeficiency disease;
- •Patients with active hepatitis B or hepatitis C (\[HBVDNA+\] or \[HCVRNA+\]); Patients with HIV infection or syphilis infection;
- •Any situations that the researchers believe will increase the risks for the subject or affect the results of the study.
Arms & Interventions
Group 1
All patients in this cohort receive daratumumab, bortezomib and dexamethasone as treatment.
All patients receive additional sFLC examination in C1D7, C1D14.
Intervention: Dara IV (Drug)
Group 1
All patients in this cohort receive daratumumab, bortezomib and dexamethasone as treatment.
All patients receive additional sFLC examination in C1D7, C1D14.
Intervention: Bortezomib (drug) (Drug)
Group 1
All patients in this cohort receive daratumumab, bortezomib and dexamethasone as treatment.
All patients receive additional sFLC examination in C1D7, C1D14.
Intervention: Dexamethasone (Drug)
Group 1
All patients in this cohort receive daratumumab, bortezomib and dexamethasone as treatment.
All patients receive additional sFLC examination in C1D7, C1D14.
Intervention: Cyclophosphamide (CTX) (Drug)
Group 1
All patients in this cohort receive daratumumab, bortezomib and dexamethasone as treatment.
All patients receive additional sFLC examination in C1D7, C1D14.
Intervention: Dara SC (Drug)
Outcomes
Primary Outcomes
Complete hematologic response
Time Frame: 6 months
Secondary Outcomes
- Overall hematologic response(6 months)
- At least one organ response(12 months)
- Minimal residual disease(6 months, 12 months)
- TTNT(12 months)
- MOD-EFS(12 months)
- MOD-PFS(12 months)
Investigators
Jin Lu, MD
Principal of Investigator
Peking University People's Hospital