Raffaella Colombatti1, Giovanni Palazzi2, Nicoletta Masera3, Lucia Dora Notarangelo4, Elisa Bonetti5, Piera Samperi6, Angelica Barone7, Silverio Perrotta8, Elena Facchini9, Maurizio Miano10, Giovanni Carlo Del Vecchio11, Maria Elena Guerzoni2, Paola Corti3, Federica Menzato1, Simone Cesaro5, Maddalena Casale8, Paolo Rigano12, Gian Luca Forni13, Giovanna Russo6, Laura Sainati1. 1. Department of Child and Maternal Health, Clinic of Pediatric Hematology Oncology, Azienda Ospedaliera-Università di Padova, Padova, Italy. 2. Department of Child and Maternal Health, Clinic of Pediatric Hematology Oncology, Azienda Ospedaliera-Università degli Studi di Modena, Modena, Italy. 3. Department of Pediatric Clinic, San Gerardo Hospital, University of Milano-Bicocca, Monza, Italy. 4. Department of Pediatric Onco-Hematology, Children's Hospital, Brescia, Italy. 5. Department of Pediatric Onco-Hematology, Azienda Ospedaliera Universitaria Integrata, Verona, Itlay. 6. Department of Pediatric Hematology and Oncology, Department of Clinical and Experimental Medicine, University of Catania, Catania, Italy. 7. Department of Pediatric Onco-Hematology, Azienda Ospedaliero-Universitaria di Parma, Parma, Italy. 8. Department of Woman, Child and General and Specialist Surgery, Università degli Studi della Campania "Luigi Vanvitelli,", Napoli, Italy. 9. Clinic of Pediatric Hematology Oncology, Policlinico S. Orsola-Malpighi, Bologna, Italy. 10. Haematology Unit, I.R.C.C.S. Istituto Giannina Gaslini, Genoa, Italy. 11. Pediatria, Policlinico di Bari, Bari, Italy. 12. Campus of Haematology Franco e Piera Cutino-A.O.O.R. "Villa Sofia-Cervello,", Palermo, Italy. 13. Hematology-Thalassemia and Congenital Anemia Center, Galliera Hospital, Genoa, Italy.
Abstract
BACKGROUND: The number of patients with sickle cell disease (SCD) has increased in Italy in the past decade due to immigration. In spite of the established efficacy of hydroxyurea (HU) in childhood, population-based data regarding its prescription and effectiveness come mainly from studies performed in adults or outside Europe. POPULATION AND METHODS: The Hydroxyurea in SCD: A Large Nation-wide Cohort Study from Italy was a retrospective cohort study of adult and pediatric patients with SCD attending 32 centers. Pediatric data are analyzed separately. RESULTS: Out of 504 children followed in 11 centers, 206 (40%) were on HU (194 SS/Sβ°, 12 SC/Sß+); 74% came from Sub-Saharian Africa and 18% from Europe. HU therapy indications for SS/Sβ° patients were as follows: 57% painful vaso-occlusive crisis, acute chest syndrome or both, 24% anemia, 8% anemia, and other reasons (the majority had Hb ≤ 8-8.5 g/dl, revealing scarce acceptance of low Hb values by pediatric hematologist). Mean starting dose was 15.5 mg/kg, and dose at full regimen was 17.1 mg/kg. Mean age at HU therapy was 7.68 years, although it was lower for SS/Sβ° patients. Only 10% started HU before 3 years. In 92%, 500 mg capsule was used; in 6%, the galenic was used; and in 2%, 100 mg tablet was used. Significant reduction of clinical events and inpatients admissions, with improvement in hematological parameters, was observed for SS/Sβ° patients and a trend toward improvement for SC/Sß+ patients was also observed. CONCLUSIONS: HU effectiveness is demonstrated in a national cohort of children with SCD living in Italy, even at a lower dose than recommended, revealing good adherence to a treatment program by a socially vulnerable group of patients such as immigrants.
BACKGROUND: The number of patients with sickle cell disease (SCD) has increased in Italy in the past decade due to immigration. In spite of the established efficacy of hydroxyurea (HU) in childhood, population-based data regarding its prescription and effectiveness come mainly from studies performed in adults or outside Europe. POPULATION AND METHODS: The Hydroxyurea in SCD: A Large Nation-wide Cohort Study from Italy was a retrospective cohort study of adult and pediatric patients with SCD attending 32 centers. Pediatric data are analyzed separately. RESULTS: Out of 504 children followed in 11 centers, 206 (40%) were on HU (194 SS/Sβ°, 12 SC/Sß+); 74% came from Sub-Saharian Africa and 18% from Europe. HU therapy indications for SS/Sβ° patients were as follows: 57% painful vaso-occlusive crisis, acute chest syndrome or both, 24% anemia, 8% anemia, and other reasons (the majority had Hb ≤ 8-8.5 g/dl, revealing scarce acceptance of low Hb values by pediatric hematologist). Mean starting dose was 15.5 mg/kg, and dose at full regimen was 17.1 mg/kg. Mean age at HU therapy was 7.68 years, although it was lower for SS/Sβ° patients. Only 10% started HU before 3 years. In 92%, 500 mg capsule was used; in 6%, the galenic was used; and in 2%, 100 mg tablet was used. Significant reduction of clinical events and inpatients admissions, with improvement in hematological parameters, was observed for SS/Sβ° patients and a trend toward improvement for SC/Sß+ patients was also observed. CONCLUSIONS: HU effectiveness is demonstrated in a national cohort of children with SCD living in Italy, even at a lower dose than recommended, revealing good adherence to a treatment program by a socially vulnerable group of patients such as immigrants.
Authors: Jifang Zhou; Jin Han; Edith A Nutescu; Victor R Gordeuk; Santosh L Saraf; Gregory S Calip Journal: Br J Haematol Date: 2018-05-16 Impact factor: 6.998
Authors: Bukola Salami; Higinio Fernandez-Sanchez; Christa Fouche; Catrin Evans; Lindiwe Sibeko; Mia Tulli; Ashley Bulaong; Stephen Owusu Kwankye; Mary Ani-Amponsah; Philomina Okeke-Ihejirika; Hayat Gommaa; Kafuli Agbemenu; Chizoma Millicent Ndikom; Solina Richter Journal: Int J Environ Res Public Health Date: 2021-03-28 Impact factor: 3.390