An update on progress of the
Cooperative Study of
Sickle Cell Disease ... for
participants and their families

Background:
Over 4000 individuals with sickle cell disease enrolled in the
project, beginning in 1978.  In 1988, only those enrolled prior
to 6 months of age or who were over age 35 years continued to be
followed, and since 1993 only the youngest group continue on the
study, which is expected to end in 1998; children in this
"Pediatric Cohort," the oldest of whom is now age 17 years, have
been participating in the CSSCD their entire lives!  A tremendous
amount of information has been recorded about participants' lives
over the years, with great effort on the part of study personnel,
participants and their families.  What have we learned so far
from this effort, and what do we hope to learn prior to
completion?

What have we learned?
- A number of CSSCD children with sickle cell anemia (SS) joined
a study which confirmed the value of twice daily penicillin in
preventing infection and death due to Streptococcus pneumoniae
(pneumococcus).  This study was a major factor in convincing
nearly all states and many foreign countries that all newborns
must be screened for sickle cell disease.  Infants can then be
identified shortly after birth to get medical care needed to
prevent death and sickness from early complications.  Many lives
have already been saved.
- Data was collected which permitted construction of "normal"
growth curves and laboratory values for children with sickle cell
disease.  Many children who seem delayed in growth or sexual
development can be reassured that they will catch up.   Anemia in
excess of what's usually seen with sickle cell disease can be
recognized and evaluated.
- Extensive analysis of the "natural history" of sickle cell
disease has occurred.  The types, frequencies and risk factors
for various complications, including (among others) pain, acute
chest syndrome (pneumonia), strokes, leg ulcers, aseptic necrosis
(chronic hip or shoulder pain) and death have been documented. 
Doctors can use this information to give their patients and
parents of newly diagnosed infants information about what to
expect for the future.  The CSSCD has also given insights as to
how to safely perform surgery and reduce risks associated with
blood transfusion.  
- It has been determined that 15% of children with sickle cell
disease have abnormal MRI scans of the brain and that some
children with sickle cell disease perform less well on
achievement testing than others without sickle cell disease;
there appears to be a relationship between the brain scan
abnormalities and achievement test results.
- There is a high incidence of lung problems in children with
sickle cell disease.  While in adults these problems are seen
particularly in those who have recurrent pneumonia, this does not
seem to be the case in children; it is not yet clear why so many
children have lung abnormalities. 

What do we hope to accomplish before the end of the study in
1998?
- Will changes on MRI of the brain and achievement test scores
change with time?  If they worsen or are associated with more
serious neurologic problems (stroke?), consideration will have to
be given to trying to intervene with some form of treatment of
affected children.
- Further clarification of abnormal performance on achievement
tests might permit specific educational help and understanding as
to why and how damage occurs.
- We need to know if lung abnormalities worsen with time, and if
so, in all or just some children.  Again, if worsening occurs
therapy has to be considered.
- Finally, treatment for sickle cell disease is best done early
before damage occurs.  We hope that by combining all the
accumulated data over the entire lives of enrolled children we
may be able to say with more certainty how well a child may do in
the future.

Also remember that:
In addition to greatly increasing the world's understanding of
sickle cell disease and improving care and survival, a number of
institutions, including our own, received financial support from
the study which kept our sickle cell clinic going.  Many of the
nurses, doctors, counselors, and laboratory personnel were able
to work with our children over the last 15 years because of the
support of the Cooperative Study.

Everyone working with the CSSCD wants to thank all the
participants and their families for being a part of the
Cooperative Study; it has been, and continues to be, a rewarding
experience.

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