LONG TERM FOLLOW-UP OF HYDROXYUREA IN SICKLE CELL DISEASELONG TERM FOLLOW-UP OF HYDROXYUREA IN SICKLE CELL DISEASE
(Abstract release date: 05/21/15)
EHA Library. Bradai M. 06/12/15; 102661; PB2001
Disclosure(s): Faculté de médecine. Unicersité Saad DahlebDeprtement de médecine
Mohamed Bradai
Contributions
Contributions
Abstract
Abstract: PB2001
Type: Publication Only
Background
Hydroxyurea (HU) is considered to be the most successful drug therapy for severe sickle cell disease (SCD) and has many characteristics of an ideal drug for sickle cell anemia (SCA) and provides therapeutic benefit through multiple mechanisms of action. HU emerged as an important therapeutic option for children and adolescents with recurrent vaso-occlusive events and sustained long-term benefits were documented
Aims
To assess long term effects of hydroxyurea therapy in infants and adults with sickle cell disease (all types)
Methods
Since 2000, there were 54 SCD patients treated with HU, 39 SS 14S/βthal and 1 SC: Mean age at inclusion was 12+/-4.4 years (4 – 22 y), just 3 patients was aged above 40y at the inclusion, sex ratio: 29F/25M. Thirty night included for standard criteria (more than 3 severe crisis pain/year and/or recurrent acute chest syndrome >1 ACS/y) and 7 because of severe chronic anemia (baseline of hemoglobin lower than 6g/dL), 2 for complex red cell allo-immunization, 1 for delayed posttransfusional hemolysis, 1 invalidant priapism, 2 association with auto-immun disease(sarcoidosis, Systemic lupus erythematosus), 1 history of stroke with transfusional difficulties, 1 for nephropathy and renal failure (creatinine 18 mg/ml). 13 patients have been splenectomized before the inclusion; Avascular Necrosis of hips was noted in 5 patients. Ferritine in 27 patients polytransfused (more 20 blood units) was 3700 ng/ml. The median daily dose of HU was 18 mg/kg (14 to 22 mg/kg/d)
Results
After a follow-up of 9 years(5 to 14 years), we observed a significant decreased (p<0,0001) of severe and recurrent acute pain crisis (5,5→0,7/y) and Acute chest syndrome (1,5→0,3/y), need of blood transfusions (8→2/year), days of hospitalization (17→3/y). Sustained clinical benefits for the most patients received long-term HU treatment. Total hemoglobin increase was 1,1g/dl (0.5 à 2.5). In the subgroup of 7 patients treated for severe anemia, the mean hemoglobin level was 6.6±0.5 g/dL prior to HU and increased to 8.2±1.5 g/dL after 1 year of therapy. The iron chelation Deferroxamine (30mg/kg/j, 3j/7) and phlebotomy, performed in 11 patients when Hb level was upper 9g/dl). We noted some severe events: acute priapism in 4 patients (1 shunt caverno-spongieux),4 hip osteonecrosis. We deplored 7 deaths: recidive of stroke (1), sepsis (1), ACS (2), progressive renal failure (1), acute hepatic sequestration (1), undetermined (1). Five were lost to follow-up after a median of 48 months. Very young patients treated have been associated with excellent growth and development. Sexual maturation, including menarche, has occurred without apparent delay. No severe side effect was related to HU treatment, which was discontinued in children mainly for transient neutropenia or thrombopenia, hypersplenism or acute splenic sequestration, followed by splenectomy(9 cases) or non-compliance (2 cases). Four women were pregnant after 8 years of HU. Long-term clinical benefits have also been observed wth improved quality of life. Ferritin levels were decreased with significant decline in group of patients treated with Deferroxamine and/or phlebotomy (3700→950 ng/ml).
Summary
Long-term exposure to HU seems associated with significantly reduced mortality, perhaps by reducing long-term end organ damage and does not appear to be associated with increased risk of secondary malignancies nor myelodysplasic syndrome. Our experience, mainly in children allows us to consider that this drug is a very potent therapeutic option for the management of severe forms of the disease, particularly in countries with limited blood supply. An updated national registry is highly needed to further assessment.
Keyword(s): Hydroxyurea, Sickle cell disease
Type: Publication Only
Background
Hydroxyurea (HU) is considered to be the most successful drug therapy for severe sickle cell disease (SCD) and has many characteristics of an ideal drug for sickle cell anemia (SCA) and provides therapeutic benefit through multiple mechanisms of action. HU emerged as an important therapeutic option for children and adolescents with recurrent vaso-occlusive events and sustained long-term benefits were documented
Aims
To assess long term effects of hydroxyurea therapy in infants and adults with sickle cell disease (all types)
Methods
Since 2000, there were 54 SCD patients treated with HU, 39 SS 14S/βthal and 1 SC: Mean age at inclusion was 12+/-4.4 years (4 – 22 y), just 3 patients was aged above 40y at the inclusion, sex ratio: 29F/25M. Thirty night included for standard criteria (more than 3 severe crisis pain/year and/or recurrent acute chest syndrome >1 ACS/y) and 7 because of severe chronic anemia (baseline of hemoglobin lower than 6g/dL), 2 for complex red cell allo-immunization, 1 for delayed posttransfusional hemolysis, 1 invalidant priapism, 2 association with auto-immun disease(sarcoidosis, Systemic lupus erythematosus), 1 history of stroke with transfusional difficulties, 1 for nephropathy and renal failure (creatinine 18 mg/ml). 13 patients have been splenectomized before the inclusion; Avascular Necrosis of hips was noted in 5 patients. Ferritine in 27 patients polytransfused (more 20 blood units) was 3700 ng/ml. The median daily dose of HU was 18 mg/kg (14 to 22 mg/kg/d)
Results
After a follow-up of 9 years(5 to 14 years), we observed a significant decreased (p<0,0001) of severe and recurrent acute pain crisis (5,5→0,7/y) and Acute chest syndrome (1,5→0,3/y), need of blood transfusions (8→2/year), days of hospitalization (17→3/y). Sustained clinical benefits for the most patients received long-term HU treatment. Total hemoglobin increase was 1,1g/dl (0.5 à 2.5). In the subgroup of 7 patients treated for severe anemia, the mean hemoglobin level was 6.6±0.5 g/dL prior to HU and increased to 8.2±1.5 g/dL after 1 year of therapy. The iron chelation Deferroxamine (30mg/kg/j, 3j/7) and phlebotomy, performed in 11 patients when Hb level was upper 9g/dl). We noted some severe events: acute priapism in 4 patients (1 shunt caverno-spongieux),4 hip osteonecrosis. We deplored 7 deaths: recidive of stroke (1), sepsis (1), ACS (2), progressive renal failure (1), acute hepatic sequestration (1), undetermined (1). Five were lost to follow-up after a median of 48 months. Very young patients treated have been associated with excellent growth and development. Sexual maturation, including menarche, has occurred without apparent delay. No severe side effect was related to HU treatment, which was discontinued in children mainly for transient neutropenia or thrombopenia, hypersplenism or acute splenic sequestration, followed by splenectomy(9 cases) or non-compliance (2 cases). Four women were pregnant after 8 years of HU. Long-term clinical benefits have also been observed wth improved quality of life. Ferritin levels were decreased with significant decline in group of patients treated with Deferroxamine and/or phlebotomy (3700→950 ng/ml).
Summary
Long-term exposure to HU seems associated with significantly reduced mortality, perhaps by reducing long-term end organ damage and does not appear to be associated with increased risk of secondary malignancies nor myelodysplasic syndrome. Our experience, mainly in children allows us to consider that this drug is a very potent therapeutic option for the management of severe forms of the disease, particularly in countries with limited blood supply. An updated national registry is highly needed to further assessment.
Keyword(s): Hydroxyurea, Sickle cell disease
Abstract: PB2001
Type: Publication Only
Background
Hydroxyurea (HU) is considered to be the most successful drug therapy for severe sickle cell disease (SCD) and has many characteristics of an ideal drug for sickle cell anemia (SCA) and provides therapeutic benefit through multiple mechanisms of action. HU emerged as an important therapeutic option for children and adolescents with recurrent vaso-occlusive events and sustained long-term benefits were documented
Aims
To assess long term effects of hydroxyurea therapy in infants and adults with sickle cell disease (all types)
Methods
Since 2000, there were 54 SCD patients treated with HU, 39 SS 14S/βthal and 1 SC: Mean age at inclusion was 12+/-4.4 years (4 – 22 y), just 3 patients was aged above 40y at the inclusion, sex ratio: 29F/25M. Thirty night included for standard criteria (more than 3 severe crisis pain/year and/or recurrent acute chest syndrome >1 ACS/y) and 7 because of severe chronic anemia (baseline of hemoglobin lower than 6g/dL), 2 for complex red cell allo-immunization, 1 for delayed posttransfusional hemolysis, 1 invalidant priapism, 2 association with auto-immun disease(sarcoidosis, Systemic lupus erythematosus), 1 history of stroke with transfusional difficulties, 1 for nephropathy and renal failure (creatinine 18 mg/ml). 13 patients have been splenectomized before the inclusion; Avascular Necrosis of hips was noted in 5 patients. Ferritine in 27 patients polytransfused (more 20 blood units) was 3700 ng/ml. The median daily dose of HU was 18 mg/kg (14 to 22 mg/kg/d)
Results
After a follow-up of 9 years(5 to 14 years), we observed a significant decreased (p<0,0001) of severe and recurrent acute pain crisis (5,5→0,7/y) and Acute chest syndrome (1,5→0,3/y), need of blood transfusions (8→2/year), days of hospitalization (17→3/y). Sustained clinical benefits for the most patients received long-term HU treatment. Total hemoglobin increase was 1,1g/dl (0.5 à 2.5). In the subgroup of 7 patients treated for severe anemia, the mean hemoglobin level was 6.6±0.5 g/dL prior to HU and increased to 8.2±1.5 g/dL after 1 year of therapy. The iron chelation Deferroxamine (30mg/kg/j, 3j/7) and phlebotomy, performed in 11 patients when Hb level was upper 9g/dl). We noted some severe events: acute priapism in 4 patients (1 shunt caverno-spongieux),4 hip osteonecrosis. We deplored 7 deaths: recidive of stroke (1), sepsis (1), ACS (2), progressive renal failure (1), acute hepatic sequestration (1), undetermined (1). Five were lost to follow-up after a median of 48 months. Very young patients treated have been associated with excellent growth and development. Sexual maturation, including menarche, has occurred without apparent delay. No severe side effect was related to HU treatment, which was discontinued in children mainly for transient neutropenia or thrombopenia, hypersplenism or acute splenic sequestration, followed by splenectomy(9 cases) or non-compliance (2 cases). Four women were pregnant after 8 years of HU. Long-term clinical benefits have also been observed wth improved quality of life. Ferritin levels were decreased with significant decline in group of patients treated with Deferroxamine and/or phlebotomy (3700→950 ng/ml).
Summary
Long-term exposure to HU seems associated with significantly reduced mortality, perhaps by reducing long-term end organ damage and does not appear to be associated with increased risk of secondary malignancies nor myelodysplasic syndrome. Our experience, mainly in children allows us to consider that this drug is a very potent therapeutic option for the management of severe forms of the disease, particularly in countries with limited blood supply. An updated national registry is highly needed to further assessment.
Keyword(s): Hydroxyurea, Sickle cell disease
Type: Publication Only
Background
Hydroxyurea (HU) is considered to be the most successful drug therapy for severe sickle cell disease (SCD) and has many characteristics of an ideal drug for sickle cell anemia (SCA) and provides therapeutic benefit through multiple mechanisms of action. HU emerged as an important therapeutic option for children and adolescents with recurrent vaso-occlusive events and sustained long-term benefits were documented
Aims
To assess long term effects of hydroxyurea therapy in infants and adults with sickle cell disease (all types)
Methods
Since 2000, there were 54 SCD patients treated with HU, 39 SS 14S/βthal and 1 SC: Mean age at inclusion was 12+/-4.4 years (4 – 22 y), just 3 patients was aged above 40y at the inclusion, sex ratio: 29F/25M. Thirty night included for standard criteria (more than 3 severe crisis pain/year and/or recurrent acute chest syndrome >1 ACS/y) and 7 because of severe chronic anemia (baseline of hemoglobin lower than 6g/dL), 2 for complex red cell allo-immunization, 1 for delayed posttransfusional hemolysis, 1 invalidant priapism, 2 association with auto-immun disease(sarcoidosis, Systemic lupus erythematosus), 1 history of stroke with transfusional difficulties, 1 for nephropathy and renal failure (creatinine 18 mg/ml). 13 patients have been splenectomized before the inclusion; Avascular Necrosis of hips was noted in 5 patients. Ferritine in 27 patients polytransfused (more 20 blood units) was 3700 ng/ml. The median daily dose of HU was 18 mg/kg (14 to 22 mg/kg/d)
Results
After a follow-up of 9 years(5 to 14 years), we observed a significant decreased (p<0,0001) of severe and recurrent acute pain crisis (5,5→0,7/y) and Acute chest syndrome (1,5→0,3/y), need of blood transfusions (8→2/year), days of hospitalization (17→3/y). Sustained clinical benefits for the most patients received long-term HU treatment. Total hemoglobin increase was 1,1g/dl (0.5 à 2.5). In the subgroup of 7 patients treated for severe anemia, the mean hemoglobin level was 6.6±0.5 g/dL prior to HU and increased to 8.2±1.5 g/dL after 1 year of therapy. The iron chelation Deferroxamine (30mg/kg/j, 3j/7) and phlebotomy, performed in 11 patients when Hb level was upper 9g/dl). We noted some severe events: acute priapism in 4 patients (1 shunt caverno-spongieux),4 hip osteonecrosis. We deplored 7 deaths: recidive of stroke (1), sepsis (1), ACS (2), progressive renal failure (1), acute hepatic sequestration (1), undetermined (1). Five were lost to follow-up after a median of 48 months. Very young patients treated have been associated with excellent growth and development. Sexual maturation, including menarche, has occurred without apparent delay. No severe side effect was related to HU treatment, which was discontinued in children mainly for transient neutropenia or thrombopenia, hypersplenism or acute splenic sequestration, followed by splenectomy(9 cases) or non-compliance (2 cases). Four women were pregnant after 8 years of HU. Long-term clinical benefits have also been observed wth improved quality of life. Ferritin levels were decreased with significant decline in group of patients treated with Deferroxamine and/or phlebotomy (3700→950 ng/ml).
Summary
Long-term exposure to HU seems associated with significantly reduced mortality, perhaps by reducing long-term end organ damage and does not appear to be associated with increased risk of secondary malignancies nor myelodysplasic syndrome. Our experience, mainly in children allows us to consider that this drug is a very potent therapeutic option for the management of severe forms of the disease, particularly in countries with limited blood supply. An updated national registry is highly needed to further assessment.
Keyword(s): Hydroxyurea, Sickle cell disease
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