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Treatment of AL amyloidosis patients with high dose melphalan and autologous stem cell transplantation produces durable remissions and improvement in quality of life



Treatment of AL amyloidosis patients with high dose melphalan and autologous stem cell transplantation produces durable remissions and improvement in quality of life



Blood 102(11): 452a-453a, November 16



AL amyloidosis (AL) is caused by a plasma cell dyscrasia in which tissue deposition of immunoglobulin light chain as amyloid fibrils leads to organ failure and death. The median survival of untreated patients is about 1 year. Treatment with oral melphalan and prednisone produces few hematologic remissions and prolongs median survival by only 3-6 months. From 1994-2002, over 700 patients with AL were seen at Boston Medical Center. Of these, 56% met criteria for treatment with HDM/SCT and 312 patients were actually treated. Treatment-related mortality was 13% and hematologic complete remissions (CR) occurred in 40% of patients evaluable at one year. Median survival of all 312 patients who initiated HDM/SCT was 4.6 years, and is estimated to be >8 yrs in those patients achieving CR. Organ function improved in 44% of evaluable patients, and in 66% of those achieving CR. Quality of life was assessed before and after treatment using the SF-36 questionnaire; 80% of the treated patients completed an initial questionnaire, and 57% of those evaluated at one year completed a followup questionnaire. At baseline, the scores (0-100) were significantly lower on all eight SF-36 sub-scales compared to age-matched population norms, except for bodily pain. For the summary scales, the mean physical component score (PCS) for the AL patients was 38 versus the norm of 48 and the mental component score (MCS) was 46 versus the norm of 51. SWOG performance score correlated well with the PCS and physical function (PF) sub-scale (r values -0.64 and -0.66, respectively). Among the SF-36 scores, and other clinical measures, PF was the best predictor of survival after HDM/SCT, with hazards ratio 0.90 per 10 additional PF units, p=0.007. At the one year followup, all SF-36 scores were improved, with the MCS reaching the population norm (mean 52 vs. norm of 51). The PCS, though improved, was still lower than normal (41 vs. norm of 48), but was greater in the subgroup of 41 patients who achieved a CR (43 vs. norm of 49). Improvement in quality of life continued during the second year for those patients that had achieved CR, rising from a baseline mean of 39 for this group to 44 at one year and 47 at two years, vs. population norm of 48. Thus, treatment of AL amyloidois patients with HDM/SCT produces durable hematologic remissions, improvements in organ function, and measurable and sustained improvements in quality of life, particularly in, but not limited to, those patients who achieve hematologic CR.

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