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Case Reports
, 91 (12), E506-E508

Organ Response in Patients With AL Amyloidosis Treated With NEOD001, an Amyloid-Directed Monoclonal Antibody

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Case Reports

Organ Response in Patients With AL Amyloidosis Treated With NEOD001, an Amyloid-Directed Monoclonal Antibody

Morie A Gertz et al. Am J Hematol.

Figures

Figure 1
Figure 1
Renal and cardiac responses of patients after NEOD001 administration. A: The urinary protein level of patient 1 (60‐year‐old man) was 5,129 mg/day at screening 40 months after he achieved CR from previous plasma cell‐directed therapy (lenalidomide and steroid, then bortezomib and lenalidomide and steroid, then high‐dose melphalan followed by autologous stem cell transplantation). After infusion 4 (16 mg/kg NEOD001), reductions in his 24‐hr proteinuria met renal response consensus criteria 4 (−36%; proteinuria 3,285 mg/day). The patient's dose was escalated to the MTD at infusion 10 (orange arrow). Best renal response was measured after 23 infusions (−88% of baseline; proteinuria 602 mg/day), and he experienced no SAEs (grade ≥3) or dose interruptions. Clinically, he experienced progressive functional improvement; edema was completely resolved, and he was no longer fatigued. B: Patient 2 (46‐year‐old man) had elevated NT‐proBNP levels (>3,000 pg/mL) both after he achieved CR from previous chemotherapy (combined cyclophosphamide, bortezomib, and dexamethasone) and at screening 9.6 months later. After infusion 8 (4 mg/kg NEOD001), his NT‐proBNP level dropped to 2196 pg/mL (−33.7% of baseline) and met consensus criteria for cardiac response 5. At infusion 14, his dose was escalated to the MTD (orange arrow), and his best response to date was recorded after 31 infusions (−72% of baseline; 929 pg/mL NT‐proBNP). The patient did experience one grade 3 SAE (chest pain), but it was deemed unrelated to NEOD001, and he has not had any dose interruptions. Clinically, he experienced progressive functional improvement and significantly improved edema with a reduction in diuretic needs. *At diagnosis, the patient had renal involvement >0.5 g/day urinary protein excretion. He achieved CR 10 months after diagnosis. CR, complete hematologic response; MTD, maximum tolerated dose; NT‐proBNP, N‐terminal fragment of probrain natriuretic peptide; SAEs, serious adverse events.

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References

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