Pomalidomide augments fetal hemoglobin production without the myelosuppressive effects of hydroxyurea in transgenic sickle cell mice.

Pharmacologic induction of fetal hemoglobin (HbF) expression is an effective treatment strategy for sickle cell disease (SCD) and β-thalassemia. Pomalidomide is a potent structural analog of thalidomide and member of a new class of immunomodulatory drugs. Recent reports demonstrated that pomalidomide reduced or eliminated transfusion requirements in certain hematologic malignancies and induced HbF ex vivo in CD34(+) progenitor cells from healthy and SCD donors.

The association between hydroxyurea treatment and pain intensity, analgesic use, and utilization in ambulatory sickle cell anemia patients.

Background: We compared daily pain, home analgesic use, and utilization among ambulatory adults in the randomized multicenter study of hydroxyurea in sickle cell anemia (MSH). We related the fetal hemoglobin (HbF) hydroxyurea response to these response variables.

Methods: Patients rated their sickle cell pain intensity (0-9), use of analgesics, and visits for pain daily.

Transfusion and chelation practices in sickle cell disease: a regional perspective.

Although most common in tropical regions, population migration has meant that sickle cell disease is now one of the most prevalent genetic diseases worldwide. The issues and challenges faced by physicians and patients have been discussed by an international group of experts representing 4 key regions: the USA, Europe, Latin America, and the Middle East/Africa. Conclusive evidence to support the use of transfusion therapy for the prevention of stroke has resulted in key changes to patient management in all regions, and increasing numbers of patients are benefiting from this management approach.

Exercise capacity and haemodynamics in patients with sickle cell disease with pulmonary hypertension treated with bosentan: results of the ASSET studies.

Doppler-defined pulmonary hypertension (PH) in sickle cell disease (SCD) is associated with 40% mortality at 40 months. To assess the effect of bosentan in SCD-PH, two randomized, double-blind, placebo-controlled, 16-week studies were initiated. Safety concerns are particularly relevant in SCD due to comorbid conditions.

Climatic and geographic temporal patterns of pain in the Multicenter Study of Hydroxyurea.

No multi-site comparisons have tested whether seasonally cold temperature or climate exacerbate pain intensity in sickle cell disease (SCD). We examined seasonal SCD pain intensity and frequency patterns and compared them with concurrent climate conditions (temperature and barometric pressure) and geography of patient residence in the Multicenter Study of Hydroxyurea (MSH). We conducted a time series analysis of the monthly average daily pain intensity (0-9 scale) and pain frequency of the 299 MSH patients from December 1991 to December 1994.

Red cell exchange in sickle cell disease.

Red cell exchange transfusions remain an effective but possibly underutilized therapy in the acute and chronic treatment of sickle cell disease. In sickle cell disease, increased blood viscosity can cause complications when the hemoglobin exceeds 10 g/dL even if this is due to simple transfusion. Red cell exchange can provide needed oxygen carrying capacity while reducing the overall viscosity of blood.