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BMJ 2011; 342 doi: https://doi.org/10.1136/bmj.d3013 (Published 18 May 2011) Cite this as: BMJ 2011;342:d3013

Adult treatment for sickle cell anaemia works for small children too

Hydroxycarbamide (previously hydroxyurea) is an established treatment for sickle cell anaemia in adults. A new trial from the US confirms that the drug is also safe and effective in children, reducing pain, dactylitis, hospital admissions, and transfusions when compared with placebo. The drug helped preserve haemoglobin concentrations, particularly fetal haemoglobin, which is thought to limit the severity of the disease.

Participants were between 9 and 18 months old when recruited. They were treated for two years. Researchers were particularly interested in the effects of hydroxycarbamide on splenic and renal function, both of which are compromised by sickle cell pathology. These results were inconclusive, but promising.

Hydroxycarbamide has a good safety profile in adults, and mild to moderate neutropenia was the only safety concern in the paediatric trial (neutrophil counts 0.5-1.25×109/L; hazard ratio 3.0, 95% CI 1.7 to 5.1). The authors reported no increase in infections among children given hydroxycarbamide.

A linked comment (p 1628) describes the findings as “extremely encouraging,” particularly for low income countries in sub-Saharan Africa, where sickle cell anaemia is common in infants and often lethal. Hydroxycarbamide is a cheap drug that, with a little international collaboration, could be made available to those who need it most.

Hard choices for women considering surgical repair of a cystocoele

Transvaginal placement of a polypropylene mesh is an increasingly popular way to repair a prolapsing anterior vaginal wall (cystocoele), and surgical practice has raced well ahead of the evidence. A rare comparative trial shows there are benefits but also serious drawbacks to the new technique. Mesh repair effectively cured more women in the short term than traditional colporrhaphy (60.8% (107/176) v 34.5% (60/174); P<0.001), but mesh placement was more complicated, took longer, and caused more blood loss (84.7 mL (SD 163.5) v 35.4 mL (35.4); P<0.001). Women treated with mesh repair kits had more trouble with …

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