Contributions
Type: Oral Presentation
Presentation during EHA20: From 14.06.2015 09:00 to 14.06.2015 09:15
Location: Room Stolz 2
Background
Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare, genetic, and life-threatening blood disease. This illness is characterized by the destruction of red blood cells and hemolytic anemia [1]. The consequent intravascular hemolysis, hypercoagulability, and bone marrow failure may result in several clinical sequels as thrombosis or renal failure [2]. Although its treatment was usually symptomatic, Eculizumab has recently shown efficient results for this disease world-wide [3] and specifically in Spanish cohorts [4].
Aims
This study was planned to describe Acute and Chronic Renal Failure (ARF and CRF) related values in PNH-patients and to assess how Eculizumab affects these parameters.
Methods
From 128 registered patients (PNH Spanish Registry; April 2014), 60 were diagnosed with classic PNH (47%). 27 of these cases (45%) also suffered ARF or CRF and were included in this retrospective and descriptive study.
Results
PNH studied patients were 12 males and 15 females with a mean age of 48.5 (±16.2) y/o whom were diagnosed with PNH when they had a mean age of 38.6 (±13.8) y/o. PNH clone values for analyzed patients was 71.7 % (±23.4) granulocytes, 76.6 % (±28.1) monocytes and 42.3 % (±23.6) erythrocytes.
All 27 patients presented renal failure. Specifically, 5 (18.5%) patients were diagnosed with CRF without any previous ARF, 8 (29.6%) were diagnosed with CRF and suffered ARF and 14 (51.9%) only presented ARF. In 20 cases, ARF diagnosis was coincident with a hemolytic crisis. In addition, a patient presented 5 ARF episodes and 4 patients presented 2 episodes.
ARF patients showed these mean values at diagnosis: 3880.3 (±2253) UI/L for LDH, 8.1 (±1.9) g/dL for Hemoglobin (Hb), 7.6% (±7.2) Reticulocytes, 117.3 (±46.7) mg/dL for Urea, 4.4 (±2.3) for Creatinine, 26.8 (±17.3) ml/min/1.73 m2 for Glomerular filtrate and 534.8 (±492.1) mg/24 h for Proteinuria. In addition, 21 out of 22 patients showed Hemoglobinuria, 15 out of 16 Hemosideruria and 9 out of 15 Urinary casts. Finally, 6 ARF patients required dialyses and there were 18 complete recoveries and 4 partial recoveries, with a mean time of 13.3 (±7.6) days. Values for CRF-diagnosed patients were 41.7 (±27.2) mg/dL for Urea, 1.3 (±0.4) mg/dL for Creatinine, 56.8 (±14.0) ml/min/1.73 m2 for Glomerular filtrate and 814.4 (±614.0) mg/24 h for Proteinuria.
After PNH diagnosis, estimated probability to evade ARF at 5 years was 56.5% (IC 95%; 35.1, 73.3) and median time without an ARF episode was estimated to be 7.3 (IC 95%; 2.2, 14.9) years. Likewise, estimated probability to avoid CRF at 5 years was 60.9% (IC 95%; 37.8, 77.7) and median time without CRF diagnosis was estimated to be 14.5 (IC 95%; 3.8, ND) years.
Finally, from 23 Eculizumab treated patients, 20 were still continuing with a mean extent of 3.9 (±2.2) years. Creatinine and Glomerular filtrate values for treated CRF patients were still very preliminary to statistical evaluation. However, recorded data showed a trend towards improvement after 18 months. Considering all treated patients, only 1 (5.3%) suffered a septic shock coincident ARF episode after treatment. All patients were alive at the end of the study.
Summary
This study described ARF and CRF values of Spanish PNH patients and estimated the probabilities to present these diseases after PNH diagnosis. Also, preliminary results suggested that there might be a low ARF incidence in Eculizumab treated patients and CRF related clinical values might have a positive development in time.
Bibliography: 1. Hillmen P et al. N Engl J Med. 1995; 2. Rachidi S et al. N Engl J Med. 2010; 3. Hillmen P et al. N Engl J Med. 2006; 4. Muñoz-Linares C et al. Eur J Haematol. 2014
Keyword(s): Acute renal failure, Chronic renal failure, PNH, Treatment
Session topic: Biology and clinics of bone marrow failure syndromes and PNH
Type: Oral Presentation
Presentation during EHA20: From 14.06.2015 09:00 to 14.06.2015 09:15
Location: Room Stolz 2
Background
Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare, genetic, and life-threatening blood disease. This illness is characterized by the destruction of red blood cells and hemolytic anemia [1]. The consequent intravascular hemolysis, hypercoagulability, and bone marrow failure may result in several clinical sequels as thrombosis or renal failure [2]. Although its treatment was usually symptomatic, Eculizumab has recently shown efficient results for this disease world-wide [3] and specifically in Spanish cohorts [4].
Aims
This study was planned to describe Acute and Chronic Renal Failure (ARF and CRF) related values in PNH-patients and to assess how Eculizumab affects these parameters.
Methods
From 128 registered patients (PNH Spanish Registry; April 2014), 60 were diagnosed with classic PNH (47%). 27 of these cases (45%) also suffered ARF or CRF and were included in this retrospective and descriptive study.
Results
PNH studied patients were 12 males and 15 females with a mean age of 48.5 (±16.2) y/o whom were diagnosed with PNH when they had a mean age of 38.6 (±13.8) y/o. PNH clone values for analyzed patients was 71.7 % (±23.4) granulocytes, 76.6 % (±28.1) monocytes and 42.3 % (±23.6) erythrocytes.
All 27 patients presented renal failure. Specifically, 5 (18.5%) patients were diagnosed with CRF without any previous ARF, 8 (29.6%) were diagnosed with CRF and suffered ARF and 14 (51.9%) only presented ARF. In 20 cases, ARF diagnosis was coincident with a hemolytic crisis. In addition, a patient presented 5 ARF episodes and 4 patients presented 2 episodes.
ARF patients showed these mean values at diagnosis: 3880.3 (±2253) UI/L for LDH, 8.1 (±1.9) g/dL for Hemoglobin (Hb), 7.6% (±7.2) Reticulocytes, 117.3 (±46.7) mg/dL for Urea, 4.4 (±2.3) for Creatinine, 26.8 (±17.3) ml/min/1.73 m2 for Glomerular filtrate and 534.8 (±492.1) mg/24 h for Proteinuria. In addition, 21 out of 22 patients showed Hemoglobinuria, 15 out of 16 Hemosideruria and 9 out of 15 Urinary casts. Finally, 6 ARF patients required dialyses and there were 18 complete recoveries and 4 partial recoveries, with a mean time of 13.3 (±7.6) days. Values for CRF-diagnosed patients were 41.7 (±27.2) mg/dL for Urea, 1.3 (±0.4) mg/dL for Creatinine, 56.8 (±14.0) ml/min/1.73 m2 for Glomerular filtrate and 814.4 (±614.0) mg/24 h for Proteinuria.
After PNH diagnosis, estimated probability to evade ARF at 5 years was 56.5% (IC 95%; 35.1, 73.3) and median time without an ARF episode was estimated to be 7.3 (IC 95%; 2.2, 14.9) years. Likewise, estimated probability to avoid CRF at 5 years was 60.9% (IC 95%; 37.8, 77.7) and median time without CRF diagnosis was estimated to be 14.5 (IC 95%; 3.8, ND) years.
Finally, from 23 Eculizumab treated patients, 20 were still continuing with a mean extent of 3.9 (±2.2) years. Creatinine and Glomerular filtrate values for treated CRF patients were still very preliminary to statistical evaluation. However, recorded data showed a trend towards improvement after 18 months. Considering all treated patients, only 1 (5.3%) suffered a septic shock coincident ARF episode after treatment. All patients were alive at the end of the study.
Summary
This study described ARF and CRF values of Spanish PNH patients and estimated the probabilities to present these diseases after PNH diagnosis. Also, preliminary results suggested that there might be a low ARF incidence in Eculizumab treated patients and CRF related clinical values might have a positive development in time.
Bibliography: 1. Hillmen P et al. N Engl J Med. 1995; 2. Rachidi S et al. N Engl J Med. 2010; 3. Hillmen P et al. N Engl J Med. 2006; 4. Muñoz-Linares C et al. Eur J Haematol. 2014
Keyword(s): Acute renal failure, Chronic renal failure, PNH, Treatment
Session topic: Biology and clinics of bone marrow failure syndromes and PNH