Sickle cell disease: time for a closer look at treatment options?

Abstract

SummaryTremendous progress has been made in the care of individuals with sickle cell over the past several decades. Major successes have been comprehensive infection prophylaxis, prediction and prevention of stroke, and better transfusion care, the latter including both prevention of alloimmunization and treatment of iron overload. However, definitive therapies remain limited to hydroxycarbamide (hydroxyurea) and stem cell transplantation, both of which have been in use for at least two decades. Despite knowing the progressive natural history of the disease with organ dysfunction, failure, and ultimately death at a young age, definitive therapies are considered for only a small proportion of individuals. Consequently, while life expectancy has improved dramatically from the last century, the ongoing pace of advancement has slowed or stalled. We believe that it is time to broaden the use of definitive therapy for those with asymptomatic disease, being cautiously more aggressive in our approach.

Reduced toxicity, myeloablative conditioning with BU, fludarabine, alemtuzumab and SCT from sibling donors in children with sickle cell disease

“…Perhaps the time has arrived where allo-SCT is offered to children with HLA-matched sibling donors with or without early SCD complications. 36,37 Limitations to this trial include the small number of patients with limited follow-up. In addition, the authors are aware that alemtuzumab is not readily available to all centers performing allo-SCT for SCD.…”

Musculoskeletal complications in sickle cell anemia patients: a ten-year retrospective review of hospital-based records (1991-2000) in two Nigerian hospitals

“…28,29 although recent studies have indicated reduced mortality rate among SCA patients, because of the upward surge in life expectancy of sickle cell patients due to better understanding and correct management. [36][37][38] In the same line recent studies have reported improvement in the life expectancy of SCA patients over the last century. 37,38 Nevertheless, sickle cell disease causes a heavy burden on the society by the high morbidity and premature death associated with it.…”

“…[36][37][38] In the same line recent studies have reported improvement in the life expectancy of SCA patients over the last century. 37,38 Nevertheless, sickle cell disease causes a heavy burden on the society by the high morbidity and premature death associated with it. 17 It was observed in the course of this study that no carrier state of the disease was admitted within the stated period of the study, which is in accordance with 39 Oni et al, that most carrier state (those with the sickle trait) does not cause clinical pathology.…”

Updated Mechanisms of Sickle Cell Disease-Associated Chronic pain

“…[26] Daily oral therapy of hydroxyurea has been demonstrated to reduce SCD associated complications including pain. [26,27] Hydroxyurea is associated with several side effects including neurological, gastrointestinal, and cutaneous side effects. [28] Additionally, hydroxyurea therapy is not a targeted treatment for SCD associated chronic pain, thus more efficient treatment options should be considered.…”