We retrospectively evaluated 55 consecutive patients who received at least one dose of
lenalidomide for relapsed/refractory
AL amyloidosis. Their median age was 63 years; 72% had heart and 75% kidney involvement and 13% were on dialysis; while 20%, 46% and 34% had Mayo stage -1, -2 and -3 disease, respectively. Median time from start of primary
therapy to
lenalidomide was 15 months (range 2-100) and median number of prior
therapies was 1 (range 1-4); 73% of the patients had prior
bortezomib and 42% were
bortezomib-refractory. On intent to treat, haematologic response rate was 51% (5.5% CRs, 20% VGPRs) and was 56% versus 40% for patients with and without prior
bortezomib and 47% versus 62.5% for
bortezomib refractory versus non-refractory patients (p = .351). Organ response was achieved by 16% of evaluable patients (22% renal, 7% liver and 3% cardiac); however, 10 (21%) patients progressed to dialysis. Median survival post
lenalidomide was 25 months.
Bortezomib-refractory patients had worse outcome (median survival of 10.5 versus 25 months for
bortezomib-sensitive patients versus not reached for
bortezomib-naive patients, p = .011). Median
lenalidomide dose was 10 mg and no patient received the 25 mg dose; however, in 60% a
dose reduction was required. Median duration of
lenalidomide therapy was 7.2 months and 46% discontinued
lenalidomide before completion of planned
therapy, mainly due to toxicity (26%) or
disease progression/no response (13%). We conclude that although
lenalidomide is a major salvage option for patients with relapsed/refractory
AL amyloidosis, its toxicity in patients with
AL amyloidosis is significant and doses should be adjusted for optimal tolerability.