ABSTRACT
Abstract Background: Treatment of sickle cell anemia is a challenging task and despite the well understood genetic and biochemical pathway of sickle hemoglobin, current therapy continues to be limited to the symptomatic treatment of pain, supplemental oxygen, antibiotics, red blood cell transfusions and hydroxyurea. SANGUINATE is a carbon monoxide releasing molecule and oxygen transfer agent under clinical development for the treatment of sickle cell anemia and comorbidities. Methods: An open-label randomized Phase Ib study was performed in adult sickle cell anemia patients. Two dose levels of SANGUINATE were compared to hydroxyurea in 24 homozygotes for Hb SS. Twelve subjects received either a low dose (160 mg/kg) of SANGUINATE or 15 mg/kg hydroxyurea. Another 12 subjects received either a high dose (320 mg/kg) of SANGUINATE or 15 mg/kg hydroxyurea. The primary endpoint was the safety of SANGUINATE versus hydroxyurea in sickle cell anemia patients. Secondary endpoints included determination of the plasma pharmacokinetics and assessment of hematologic measurements. Results: Musculoskeletal related adverse events were the most common. Transient troponin I levels increased in three patients, one of whom had an increase in tricuspid regurgitant velocity; however, no clinical signs were noted. Following an assessment of vital signs, tricuspid regurgitant velocity, electrocardiogram, serum biochemistry, hematology, urinalysis, and analysis of reported adverse events, SANGUINATE was found to be safe in stable sickle cell anemia patients. Conclusions: The clinical trial met its primary objective of demonstrating an acceptable safety profile for SANGUINATE in patients with sickle cell anemia. This trial established the safety of SANGUINATE at both dose levels and permitted its advance to Phase II trials.
Subject(s)
Humans , Male , Female , Adult , Comorbidity , Randomized Controlled Trial , Anemia, Sickle Cell/therapyABSTRACT
Dada la reciente profusión de noticias, comentarios y propuestas acerca de una nueva ''Reforma a la salud'' se presentan unas breves reflexiones sobre algunos aspectos relacionados con el tema, en las que se incluyen: consideraciones sobre algunos asuntos semánticos de la Ley 100, la importancia que para mejorar la salud de la población tienen los determinantes sociales de la salud ajenos a la medicina, el escenario contradictorio y costoso que se instauró con el actual sistema y los inconvenientes derivados de la medicalización que caracteriza nuestro sistema de Seguridad Social, pues hacen que este descuide otros de sus componentes importantes.
In view of the overwhelming amount of news, statements and proposals concerning the new Colombian ''Health Reform'', some commentaries are presented in this article regarding conflictive semantic issues of the law that created the Colombian Social Security System, the impact of different social issues on public health, the contradictory and expensive scenario of the current Colombian health system, and the improvisation and difficulties in implementing such law, which have contributed to neglect in other important aspects of the Social Security System.