Stories about: outcomes

A double-shot of good news for SCID: Promising transplant and gene therapy data

Hematopoietic hierarchy aging blood cell hematopoietic stem cell blood disorder Derrick Rossi
Blood-forming hematopoietic stem cells (top) give rise to all blood and immune cell types. In children with SCID, the steps leading to immune cells are broken.

In the world of fatal congenital immunodeficiency diseases, good news is always welcome, because most patients die before their first birthday if not treated. Babies with severe combined immunodeficiency disease, aka SCID or the “bubble boy disease,” now have more hope for survival thanks to two pieces of good news.

Transplants are looking up

First came a July paper in the New England Journal of Medicine (NEJM) by the Primary Immune Deficiency Treatment Consortium. This North American collaborative analyzed a decade’s worth of outcomes of hematopoietic stem cell transplant (HSCT), currently the only standard treatment option for SCID that has a chance of providing a permanent cure.

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Does it get better? Surgical outcomes in children with cerebral palsy

Children with severe cerebral palsy sometimes need surgery to help their hips and spine. But do these operations actually improve quality-of-life? Rachel DiFazio would like to know. (PaulEisenberg/Flickr)

Children with cerebral palsy (CP), the most common form of physical disability in children, all experience at least some difficulties in communication and movement. Those with the most severe forms of CP sometimes undergo reconstructive surgery on their hips and spine to correct dislocations or scoliosis. But do these operations actually improve quality of life?

“I’ve taken care of children with cerebral palsy for 21 years, and I’ve always wondered what the outcomes were of the surgeries,” says Rachel DiFazio, a nurse practitioner with the CP Program at Children’s Hospital Boston. “We have a lot of X-ray data and range-of-motion data, but we don’t really know if it gets any easier to take care of these children, whether life gets a little bit easier after the surgery, and in what ways.”

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