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Long-shot stem-cell treatment gives two brothers a future


2 Star

Long-shot stem-cell treatment gives two brothers a future

Our Review Summary

This was a well-written story from the human interest perspective. The disorder is not well known and there are currently no effective treatments.  The potential benefit offered by the transplant program is significant but the potential downsides are as well.  Facts are missing related to potential harms, the small number of cases performed to date and limited follow up.  This leaves the reader with the impression that this experimental approach is a cure for the disorder.  While we can all hope that impression is valid, hope should not cloud the reality that it is too soon to tell the value of the approach, much less to label it a cure. The words used in a story like this carry a great deal of weight.

It was a mistake to print without challenge the investigator’s claims about taking “one more disorder off the incurable list” and labeling this a “home run.”   He may feel that way.  But the story should have provided some independent expert perspective on these claims.  Maybe others would feel that way as well.  But the story doesn’t help us know that.  We only get the conflicted view of the investigator.  


Does the story adequately discuss the costs of the intervention?

Not Applicable

There were no cost estimates or mention of whether the treatment would be paid for by insurance.  However as the story was clear that the approach was experimental, perhaps it is too early to expect information about costs. It would have been ideal, however, for the report to have noted the significant cost of a bone marrow transplant and note the likelihood of similar costs with this procedure.

Does the story adequately quantify the benefits of the treatment/test/product/procedure?

Not Satisfactory

The benefits of the experimental approach were heralded as a cure.  However, it is too soon to know whether it is a cure or a treatment that provides a temporary respite from disease complications. 

Does the story adequately explain/quantify the harms of the intervention?

Not Satisfactory

There was no mention of possible harms associated with the treatment.  The potential harms related to a bone marrow transplant are significant and all were ignored in the story.  While the disease is horrible, bone marrow transplantation is a major process with a signifcant potential for major side effects and a lack of guarantee of success.  

The story mentioned that the older brother of the featured child is soon to be treated.  Are there additional complications when the treatment is used in older children?  Or for individuals that don’t have a "perfect match"?  The harms associated with treatment in such cases also should have been addressed.

Does the story seem to grasp the quality of the evidence?

Not Satisfactory

The evidence presented in this story inflated the outcomes which have been observed to date.  The story referred to the experimental approach as a cure for the disease, when it is really premature to make this claim.  Although the little boy who was treated currently has had wonderful resolution of some of the symptoms of the disorder, which is terrific – symptom resolution in one child does not constitute a cure.  It also remains to be seen whether this will be a long term fix for the disorder, even in this child. So, while the resolution of symptoms in this child is good news, the jury is still out on the final value of this experimental approach. 

Does the story commit disease-mongering?


The story did not engage in disease mongering; it made clear that the disease being discussed was rare and its cause was of genetic origin.

Does the story use independent sources and identify conflicts of interest?

Not Satisfactory

All of the medical professionals quoted in this story are involved in the specific case reported on.  Comments from experts in this disease and/or long term effects of bone marrow transplantation would have provided much needed balance.

Does the story compare the new approach with existing alternatives?

Not Satisfactory

There was no discussion of treatments currently used in the management of this condition. A simple statement noting that all current treatments simply treat symptoms would have sufficed.  The DebRA website lists two clinical trials so there appears to be additional research interest in this disorder. 

Does the story establish the availability of the treatment/test/product/procedure?

Not Satisfactory

The story reported on the experimental approach used for the featured child as though it were part of an active clinical trial.  It would have been very easy to have included information about the trial for those who might be interested.  If the investigators have only recruited 3 of the planned 30 participants, it would have been useful to include info about access to the trial.

Does the story establish the true novelty of the approach?


This appears to be a novel experimental approach to this disease.

Does the story appear to rely solely or largely on a news release?

Not Applicable

We can’t be sure if the story relied largely on a news release.  The University did issue a news release on 6/3/08 and an earlier one on Nov. 1, 2007.  We do know that the story didn’t quote any independent sources.

Total Score: 2 of 8 Satisfactory


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