AL AMYLOIDOSIS: REAL WORLD EVIDENCE FROM ARGENTINA
(Abstract release date: 05/19/16)
EHA Library. NUCIFORA E. 06/09/16; 134901; PB2001
Dr. ELSA NUCIFORA
Contributions
Contributions
Abstract
Abstract: PB2001
Type: Publication Only
Background
AL amyloidosis is a challenge in clinical practice due to the difficulties in diagnosis and treatment. We present our experience from the RIA Amyloidosis Registry managing this patients.
Aims
To evaluate the characteristics and outcomes of patients with Amyloidosis in Argentina.
Methods
Ambispective cohort observational study of patients with AL Amyloidosis included in the Amyloidosis RIA Registry from January 2006 till July 2015. Review of electronic medical records by the amyloidosis team.Data were analysed with SPSS program using conventional descriptive statistics and Kaplan Meier survival curves.
Results
The RIA Registry includes 140 patients: 6% (9) AA amyloidosis, 7% (10) FAP amyloidosis; 8%(11) senil amyloidosis; 24% (36) localized amyloidosis; 18% (27) unidentified and 34% (47) AL amyloidosis. Multiple myeloma patients with amyloidosis were excluded from the Registry.We included in this study the 47 patients with confirmed AL amyloidosis. 64% (31) were males with a median age of 58 years (35-81). The main reasons for consultation were heart failure (81%); proteinuria (62%); nephrotic syndrome (30%); renal failure (26); peripheral neuropathy; autonomic disfunction and gastrointestinal complaints (10%).In the laboratory workup only 56% of patients had a measurable M protein spike. Nevertheless, all patients had detectable clonal free light chains by inmunofixation or serum free light chain assays. The most frecuente light chain involved was lambda. Data regarding proBNP or BNP were available in 34 patients and only 4 patients had normal results.Due to delay in the diagnosis 9 patients died before starting proper treatment. Other 6 patients were treated in other hospitals (no data available). From the 32 patients treated in our hospital: 5 patients received upfront cardiac transplantation followed by CYBORD; 20 patients received low dose CYBORD; 4 patients received IMIDs and 3 patients conventional chemotherapy (prior to 2008). Autologous bone marrow transplantation was performed in 10 patients. The response rate was: complete response in 18 patients; partial reponse in 7 patients; progressive disease 1 patient; not evaluable 6 patients. There were 7 deaths; 3 in complete reponse and 2 in partial response. The main causes of death were cardiac failure (5) and sepsis (2).The median overall survival of patients treated in our hospital was 86 months (IC95 27 – 144).
Conclusion
AL Amyloidosis is underdiagnosed in Argentina. The consequent delay in diagnosis and start of treatment has a huge impact in the quality of life and survival of this patients. Low dose CYBORD is a good treatment option with and adecuate response rate and safety profile considering the difficulties associated with amyloid organ damage. M spike and free light chains response do not always correlates with organ response which usually occurs much later. Therefore it is important to consider heart transplantation in young patients with severe amyloid myocardiopathy as it remains the main cause of death in this patients.
Session topic: E-poster
Type: Publication Only
Background
AL amyloidosis is a challenge in clinical practice due to the difficulties in diagnosis and treatment. We present our experience from the RIA Amyloidosis Registry managing this patients.
Aims
To evaluate the characteristics and outcomes of patients with Amyloidosis in Argentina.
Methods
Ambispective cohort observational study of patients with AL Amyloidosis included in the Amyloidosis RIA Registry from January 2006 till July 2015. Review of electronic medical records by the amyloidosis team.Data were analysed with SPSS program using conventional descriptive statistics and Kaplan Meier survival curves.
Results
The RIA Registry includes 140 patients: 6% (9) AA amyloidosis, 7% (10) FAP amyloidosis; 8%(11) senil amyloidosis; 24% (36) localized amyloidosis; 18% (27) unidentified and 34% (47) AL amyloidosis. Multiple myeloma patients with amyloidosis were excluded from the Registry.We included in this study the 47 patients with confirmed AL amyloidosis. 64% (31) were males with a median age of 58 years (35-81). The main reasons for consultation were heart failure (81%); proteinuria (62%); nephrotic syndrome (30%); renal failure (26); peripheral neuropathy; autonomic disfunction and gastrointestinal complaints (10%).In the laboratory workup only 56% of patients had a measurable M protein spike. Nevertheless, all patients had detectable clonal free light chains by inmunofixation or serum free light chain assays. The most frecuente light chain involved was lambda. Data regarding proBNP or BNP were available in 34 patients and only 4 patients had normal results.Due to delay in the diagnosis 9 patients died before starting proper treatment. Other 6 patients were treated in other hospitals (no data available). From the 32 patients treated in our hospital: 5 patients received upfront cardiac transplantation followed by CYBORD; 20 patients received low dose CYBORD; 4 patients received IMIDs and 3 patients conventional chemotherapy (prior to 2008). Autologous bone marrow transplantation was performed in 10 patients. The response rate was: complete response in 18 patients; partial reponse in 7 patients; progressive disease 1 patient; not evaluable 6 patients. There were 7 deaths; 3 in complete reponse and 2 in partial response. The main causes of death were cardiac failure (5) and sepsis (2).The median overall survival of patients treated in our hospital was 86 months (IC95 27 – 144).
Conclusion
AL Amyloidosis is underdiagnosed in Argentina. The consequent delay in diagnosis and start of treatment has a huge impact in the quality of life and survival of this patients. Low dose CYBORD is a good treatment option with and adecuate response rate and safety profile considering the difficulties associated with amyloid organ damage. M spike and free light chains response do not always correlates with organ response which usually occurs much later. Therefore it is important to consider heart transplantation in young patients with severe amyloid myocardiopathy as it remains the main cause of death in this patients.
Session topic: E-poster
Abstract: PB2001
Type: Publication Only
Background
AL amyloidosis is a challenge in clinical practice due to the difficulties in diagnosis and treatment. We present our experience from the RIA Amyloidosis Registry managing this patients.
Aims
To evaluate the characteristics and outcomes of patients with Amyloidosis in Argentina.
Methods
Ambispective cohort observational study of patients with AL Amyloidosis included in the Amyloidosis RIA Registry from January 2006 till July 2015. Review of electronic medical records by the amyloidosis team.Data were analysed with SPSS program using conventional descriptive statistics and Kaplan Meier survival curves.
Results
The RIA Registry includes 140 patients: 6% (9) AA amyloidosis, 7% (10) FAP amyloidosis; 8%(11) senil amyloidosis; 24% (36) localized amyloidosis; 18% (27) unidentified and 34% (47) AL amyloidosis. Multiple myeloma patients with amyloidosis were excluded from the Registry.We included in this study the 47 patients with confirmed AL amyloidosis. 64% (31) were males with a median age of 58 years (35-81). The main reasons for consultation were heart failure (81%); proteinuria (62%); nephrotic syndrome (30%); renal failure (26); peripheral neuropathy; autonomic disfunction and gastrointestinal complaints (10%).In the laboratory workup only 56% of patients had a measurable M protein spike. Nevertheless, all patients had detectable clonal free light chains by inmunofixation or serum free light chain assays. The most frecuente light chain involved was lambda. Data regarding proBNP or BNP were available in 34 patients and only 4 patients had normal results.Due to delay in the diagnosis 9 patients died before starting proper treatment. Other 6 patients were treated in other hospitals (no data available). From the 32 patients treated in our hospital: 5 patients received upfront cardiac transplantation followed by CYBORD; 20 patients received low dose CYBORD; 4 patients received IMIDs and 3 patients conventional chemotherapy (prior to 2008). Autologous bone marrow transplantation was performed in 10 patients. The response rate was: complete response in 18 patients; partial reponse in 7 patients; progressive disease 1 patient; not evaluable 6 patients. There were 7 deaths; 3 in complete reponse and 2 in partial response. The main causes of death were cardiac failure (5) and sepsis (2).The median overall survival of patients treated in our hospital was 86 months (IC95 27 – 144).
Conclusion
AL Amyloidosis is underdiagnosed in Argentina. The consequent delay in diagnosis and start of treatment has a huge impact in the quality of life and survival of this patients. Low dose CYBORD is a good treatment option with and adecuate response rate and safety profile considering the difficulties associated with amyloid organ damage. M spike and free light chains response do not always correlates with organ response which usually occurs much later. Therefore it is important to consider heart transplantation in young patients with severe amyloid myocardiopathy as it remains the main cause of death in this patients.
Session topic: E-poster
Type: Publication Only
Background
AL amyloidosis is a challenge in clinical practice due to the difficulties in diagnosis and treatment. We present our experience from the RIA Amyloidosis Registry managing this patients.
Aims
To evaluate the characteristics and outcomes of patients with Amyloidosis in Argentina.
Methods
Ambispective cohort observational study of patients with AL Amyloidosis included in the Amyloidosis RIA Registry from January 2006 till July 2015. Review of electronic medical records by the amyloidosis team.Data were analysed with SPSS program using conventional descriptive statistics and Kaplan Meier survival curves.
Results
The RIA Registry includes 140 patients: 6% (9) AA amyloidosis, 7% (10) FAP amyloidosis; 8%(11) senil amyloidosis; 24% (36) localized amyloidosis; 18% (27) unidentified and 34% (47) AL amyloidosis. Multiple myeloma patients with amyloidosis were excluded from the Registry.We included in this study the 47 patients with confirmed AL amyloidosis. 64% (31) were males with a median age of 58 years (35-81). The main reasons for consultation were heart failure (81%); proteinuria (62%); nephrotic syndrome (30%); renal failure (26); peripheral neuropathy; autonomic disfunction and gastrointestinal complaints (10%).In the laboratory workup only 56% of patients had a measurable M protein spike. Nevertheless, all patients had detectable clonal free light chains by inmunofixation or serum free light chain assays. The most frecuente light chain involved was lambda. Data regarding proBNP or BNP were available in 34 patients and only 4 patients had normal results.Due to delay in the diagnosis 9 patients died before starting proper treatment. Other 6 patients were treated in other hospitals (no data available). From the 32 patients treated in our hospital: 5 patients received upfront cardiac transplantation followed by CYBORD; 20 patients received low dose CYBORD; 4 patients received IMIDs and 3 patients conventional chemotherapy (prior to 2008). Autologous bone marrow transplantation was performed in 10 patients. The response rate was: complete response in 18 patients; partial reponse in 7 patients; progressive disease 1 patient; not evaluable 6 patients. There were 7 deaths; 3 in complete reponse and 2 in partial response. The main causes of death were cardiac failure (5) and sepsis (2).The median overall survival of patients treated in our hospital was 86 months (IC95 27 – 144).
Conclusion
AL Amyloidosis is underdiagnosed in Argentina. The consequent delay in diagnosis and start of treatment has a huge impact in the quality of life and survival of this patients. Low dose CYBORD is a good treatment option with and adecuate response rate and safety profile considering the difficulties associated with amyloid organ damage. M spike and free light chains response do not always correlates with organ response which usually occurs much later. Therefore it is important to consider heart transplantation in young patients with severe amyloid myocardiopathy as it remains the main cause of death in this patients.
Session topic: E-poster
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