Contributions
Abstract: PB1657
Type: Publication Only
Session title: Myeloma and other monoclonal gammopathies - Clinical
Background
Light chain amyloidosis (AL) is a rare but severe disease due to deposition of misfolded protein in tissues leading to progressive organ failure. Its prognosis is mostly due to the degree of cardiac involvement at diagnosis. In the past years, thanks to the introduction of new agents, such as Daratumumab, a huge improvement of the prognosis has been observed due to better efficacy and less toxicity as well.
Aims
This retrospective review aimed to evaluate the reported improvement in a real life population. For this purpose, we looked at our entire AL patients population during the past 15 years.
Methods
We collected the data of 42 AL patients from two hospitals in Brussels from 2006 until 2019 and separated them into two cohorts: 2006-2015 and 2016-2019, before and after the introduction of Daratumumab. We looked into and then compared baseline characteristics, treatments, overall survival (OS), response rate, toxicities but also time from first symptoms to diagnosis between the two cohorts.
Results
The median age at diagnosis was 62.3 62 (58-72) years, 45% were females, and 23% of patients had an ECOG performance status ≥ 3. Basics characteristics of the two groups were comparable no difference regarding the number of organ involvement and their severity (stage 4 revised Mayo Clinic 23 vs 25% respectively, p=1). No improvement concerning the time to establish the diagnosis from first symptoms described by patients was observed (109,5 months versus 181 months, p=0.3) between the two periods of time. In the second cohort, an increase of Bortezomib-based regimens use was observed (71 vs 59%) and 12 patients were treated by Daratumumab in the relapse setting. The hematological overall RR was 65%, with high-quality response (≥ very good partial remission)in 41% of the patients. There was no difference in the RR between the two periods. Median OS was 33 months for the whole cohort. However, an improvement in overall survival is neat between the two cohorts with median OS (mOS) of 7 months for the first period of time compared to a mOS that was not reached yet in the second cohort (p=0.01). As expected, concerning the whole population, the mOS was significantly and hugly better in patients with PS 0-2 comparing to those with PS 3-4 (116 vs 8,9 months; p=0,009.). Moreover, median mOS according to stage of Mayo Clinic was 55,1 months for stage 1-2 and 32,1 months for stage 3-4 (HR 0.58). Within the specific population treated by Daratumumab(N=12) , we described a significant improvement of mOS compared to other treatments (7 months versus not reached; p=0,01). Moreover, less toxicities were described with 25% of infectious and 17% of haematological complications of > grade 2.
Conclusion
We observed a significant improvement in overall survival as described in other recent and larger publications. These encouraging results are mostly due to the introduction of new agents, especially Daratumumab in the relapse setting. Also, new guidelines recommending to switch treatment quickly if no satisfactory response is reached, are part of this success. However, even if a massive effort has been done in the recent past years by scientific authorities to improve the time to diagnosis (recommendation of MGUS screening i.e.), no difference was observed in the time needed to establish the diagnosis and hence, in the amount of severe cases at diagnosis between the two periods of time.
Finally, according to recent studies, Daratumumab is well tolerated and extremely efficient in AL amyloidosis.
Keyword(s): AL amyloidosis, Survival, Toxicity, Treatment
Abstract: PB1657
Type: Publication Only
Session title: Myeloma and other monoclonal gammopathies - Clinical
Background
Light chain amyloidosis (AL) is a rare but severe disease due to deposition of misfolded protein in tissues leading to progressive organ failure. Its prognosis is mostly due to the degree of cardiac involvement at diagnosis. In the past years, thanks to the introduction of new agents, such as Daratumumab, a huge improvement of the prognosis has been observed due to better efficacy and less toxicity as well.
Aims
This retrospective review aimed to evaluate the reported improvement in a real life population. For this purpose, we looked at our entire AL patients population during the past 15 years.
Methods
We collected the data of 42 AL patients from two hospitals in Brussels from 2006 until 2019 and separated them into two cohorts: 2006-2015 and 2016-2019, before and after the introduction of Daratumumab. We looked into and then compared baseline characteristics, treatments, overall survival (OS), response rate, toxicities but also time from first symptoms to diagnosis between the two cohorts.
Results
The median age at diagnosis was 62.3 62 (58-72) years, 45% were females, and 23% of patients had an ECOG performance status ≥ 3. Basics characteristics of the two groups were comparable no difference regarding the number of organ involvement and their severity (stage 4 revised Mayo Clinic 23 vs 25% respectively, p=1). No improvement concerning the time to establish the diagnosis from first symptoms described by patients was observed (109,5 months versus 181 months, p=0.3) between the two periods of time. In the second cohort, an increase of Bortezomib-based regimens use was observed (71 vs 59%) and 12 patients were treated by Daratumumab in the relapse setting. The hematological overall RR was 65%, with high-quality response (≥ very good partial remission)in 41% of the patients. There was no difference in the RR between the two periods. Median OS was 33 months for the whole cohort. However, an improvement in overall survival is neat between the two cohorts with median OS (mOS) of 7 months for the first period of time compared to a mOS that was not reached yet in the second cohort (p=0.01). As expected, concerning the whole population, the mOS was significantly and hugly better in patients with PS 0-2 comparing to those with PS 3-4 (116 vs 8,9 months; p=0,009.). Moreover, median mOS according to stage of Mayo Clinic was 55,1 months for stage 1-2 and 32,1 months for stage 3-4 (HR 0.58). Within the specific population treated by Daratumumab(N=12) , we described a significant improvement of mOS compared to other treatments (7 months versus not reached; p=0,01). Moreover, less toxicities were described with 25% of infectious and 17% of haematological complications of > grade 2.
Conclusion
We observed a significant improvement in overall survival as described in other recent and larger publications. These encouraging results are mostly due to the introduction of new agents, especially Daratumumab in the relapse setting. Also, new guidelines recommending to switch treatment quickly if no satisfactory response is reached, are part of this success. However, even if a massive effort has been done in the recent past years by scientific authorities to improve the time to diagnosis (recommendation of MGUS screening i.e.), no difference was observed in the time needed to establish the diagnosis and hence, in the amount of severe cases at diagnosis between the two periods of time.
Finally, according to recent studies, Daratumumab is well tolerated and extremely efficient in AL amyloidosis.
Keyword(s): AL amyloidosis, Survival, Toxicity, Treatment