TY - JOUR
T1 - Lenalidomide, cyclophosphamide, and dexamethasone (CRd) for light-chain amyloidosis
T2 - Long-term results from a phase 2 trial
AU - Kumar, Shaji K.
AU - Hayman, Suzanne R.
AU - Buadi, Francis K.
AU - Roy, Vivek
AU - Lacy, Martha Q.
AU - Gertz, Morie A.
AU - Allred, Jacob
AU - Laumann, Kristina M.
AU - Bergsagel, Leif P.
AU - Dingli, David
AU - Mikhael, Joseph R.
AU - Reeder, Craig B.
AU - Stewart, A. Keith
AU - Zeldenrust, Steven R.
AU - Greipp, Philip R.
AU - Lust, John A.
AU - Fonseca, Rafael
AU - Russell, Stephen J.
AU - Rajkumar, S. Vincent
AU - Dispenzieri, Angela
PY - 2012/5/24
Y1 - 2012/5/24
N2 - Light-chain (AL) amyloidosis remains incurable despite recent therapeutic advances. Given the activity of the lenalidomidealkylating agent combination in myeloma, we designed this phase 2 trial of lenalidomide, cyclophosphamide, and dexamethasone in AL amyloidosis. Thirty-five patients, including 24 previously untreated, were enrolled. Nearly one-half of the patients had cardiac stage III disease and 28% had ≥ 3 organs involved. The overall hematologic response (≥ partial response [PR]) rate was 60%, including 40% with very-good partial response or better. Using serum-free light chain for assessing response, 77% of patients had a hematologic response. Organ responses were seen in 29% of patients and were limited to those with a hematologic response. The median hematologic progressionfree survival was 28.3 months, and the median overall survival was 37.8 months. Hematologic toxicity was the predominant adverse event, followed by fatigue, edema, and gastrointestinal symptoms. A grade 3 or higher toxicity occurred in 26 patients (74%) including ≥ grade 3 hematologic toxicity in 16 patients (46%) and ≥ grade 3 nonhematologic toxicity in 25 patients (71%). Seven patients (20%) died on study, primarily because of advanced disease. Lenalidomide, cyclophosphamide, and dexamethasone (CRd) is an effective combination for treatment of AL amyloidosis and leads to durable hematologic responses as well as organ responses with manageable toxicity. The trial was registered at www.clinicaltrials.gov (NCT00564889).
AB - Light-chain (AL) amyloidosis remains incurable despite recent therapeutic advances. Given the activity of the lenalidomidealkylating agent combination in myeloma, we designed this phase 2 trial of lenalidomide, cyclophosphamide, and dexamethasone in AL amyloidosis. Thirty-five patients, including 24 previously untreated, were enrolled. Nearly one-half of the patients had cardiac stage III disease and 28% had ≥ 3 organs involved. The overall hematologic response (≥ partial response [PR]) rate was 60%, including 40% with very-good partial response or better. Using serum-free light chain for assessing response, 77% of patients had a hematologic response. Organ responses were seen in 29% of patients and were limited to those with a hematologic response. The median hematologic progressionfree survival was 28.3 months, and the median overall survival was 37.8 months. Hematologic toxicity was the predominant adverse event, followed by fatigue, edema, and gastrointestinal symptoms. A grade 3 or higher toxicity occurred in 26 patients (74%) including ≥ grade 3 hematologic toxicity in 16 patients (46%) and ≥ grade 3 nonhematologic toxicity in 25 patients (71%). Seven patients (20%) died on study, primarily because of advanced disease. Lenalidomide, cyclophosphamide, and dexamethasone (CRd) is an effective combination for treatment of AL amyloidosis and leads to durable hematologic responses as well as organ responses with manageable toxicity. The trial was registered at www.clinicaltrials.gov (NCT00564889).
UR - http://www.scopus.com/inward/record.url?scp=84861521224&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84861521224&partnerID=8YFLogxK
U2 - 10.1182/blood-2012-01-407791
DO - 10.1182/blood-2012-01-407791
M3 - Article
C2 - 22504925
AN - SCOPUS:84861521224
SN - 0006-4971
VL - 119
SP - 4860
EP - 4867
JO - Blood
JF - Blood
IS - 21
ER -