Contributions
Abstract: PB2147
Type: Publication Only
Background
Hydroxyurea (HU) has lately been used in the treatment of patients with severe sickle cell disease (SCD). Despite documented benefits on laboratory and clinical parameters in SCD patients, there are few reports about drug’s long-term safety and efficacy in pediatric patients with SCD – even more so in the rare patient subgroup of sickle/beta thalassemia.
Aims
A prospective, long term evaluation of HU efficacy and safety in children and adolescents with sickle/beta thalassemia (S/b thal).
Methods
Results
A significant reduction in vaso-occlusive crises as compared to prior to HU treatment was noted (median: 1 episode per year before HU, range: 0-2.5 vs median: 0.24 episodes per study year after HU, range: 0-1.33, p=0.011). A significant reduction in hospitaliizations was also reported (median: 1 per year before HU, range: 0.3-2 vs median: 0.16 per study year after HU, 0-0.83, p=0.005). None of the patients presented with severe clinical events such as acute chest syndrome, avascular bone necrosis, stroke or splenic sequestration during the study period. With regards to hematological parameters, a significant increase in HbF (10.2±6.5% vs 16.6±7.1% p=0.02), MCV (66.1±3.9fl vs 79.3±8.4fl, p<0.001) and MCH (20.9±1.2pg vs 25.3±2.2pg, p<0.001), as well as a decrease in reticulocyte count (7.7 ± 3.3% vs 5.0 ± 1.9%, p=0.039), WBC count (9,566 ± 3,674/μl, vs 7,466 ± 3,460/μl, p=0.009) and PLT count (333,778/μl, ± 170,227 vs 272,111 ± 160,304/μl, p=0.007) was noted. Concerning adverse events, one patient presented with mild transaminasemia, one with mild elevation of serum creatinine levels and one with pancytopenia. Due to persistent pancytopenia HU treatment was discontinued in the last mentioned patient, but was restarted a year later due to frequent vaso-occlusive events - despite the patient being put on transfusions after initial HU discontinuation. Besides the pancytopenia episode, the rest of the mentioned toxicities were short-term and dose-dependant.
Conclusion
The study indicates that HU has an overall safe profile and results in a marked improvement of clinical course in pediatric S/b thal patients.
Session topic: 25. Sickle cell disease
Keyword(s): sickle cell disease, Hydroxyurea, Childhood
Abstract: PB2147
Type: Publication Only
Background
Hydroxyurea (HU) has lately been used in the treatment of patients with severe sickle cell disease (SCD). Despite documented benefits on laboratory and clinical parameters in SCD patients, there are few reports about drug’s long-term safety and efficacy in pediatric patients with SCD – even more so in the rare patient subgroup of sickle/beta thalassemia.
Aims
A prospective, long term evaluation of HU efficacy and safety in children and adolescents with sickle/beta thalassemia (S/b thal).
Methods
Results
A significant reduction in vaso-occlusive crises as compared to prior to HU treatment was noted (median: 1 episode per year before HU, range: 0-2.5 vs median: 0.24 episodes per study year after HU, range: 0-1.33, p=0.011). A significant reduction in hospitaliizations was also reported (median: 1 per year before HU, range: 0.3-2 vs median: 0.16 per study year after HU, 0-0.83, p=0.005). None of the patients presented with severe clinical events such as acute chest syndrome, avascular bone necrosis, stroke or splenic sequestration during the study period. With regards to hematological parameters, a significant increase in HbF (10.2±6.5% vs 16.6±7.1% p=0.02), MCV (66.1±3.9fl vs 79.3±8.4fl, p<0.001) and MCH (20.9±1.2pg vs 25.3±2.2pg, p<0.001), as well as a decrease in reticulocyte count (7.7 ± 3.3% vs 5.0 ± 1.9%, p=0.039), WBC count (9,566 ± 3,674/μl, vs 7,466 ± 3,460/μl, p=0.009) and PLT count (333,778/μl, ± 170,227 vs 272,111 ± 160,304/μl, p=0.007) was noted. Concerning adverse events, one patient presented with mild transaminasemia, one with mild elevation of serum creatinine levels and one with pancytopenia. Due to persistent pancytopenia HU treatment was discontinued in the last mentioned patient, but was restarted a year later due to frequent vaso-occlusive events - despite the patient being put on transfusions after initial HU discontinuation. Besides the pancytopenia episode, the rest of the mentioned toxicities were short-term and dose-dependant.
Conclusion
The study indicates that HU has an overall safe profile and results in a marked improvement of clinical course in pediatric S/b thal patients.
Session topic: 25. Sickle cell disease
Keyword(s): sickle cell disease, Hydroxyurea, Childhood