Prevalence of Diseases and Conditions Amenable to Stem Cell Transplant or Infusion Among Families Storing Newborn Stem Cells at a Large Private Cord Blood Bank

Background:  Little is known about the prevalence of diseases amenable to allogeneic or autologous stem cell infusions among families storing newborn stem cells in private cord blood banks.

Methods:  We surveyed families with at least one child who had stored newborn stem cells at the largest private cord blood bank in the US.  We requested that they complete a Family Health Questionnaire to determine whether a child with stored stem cells or a first degree relative of that child currently had one or more of 16 diseases or conditions amenable primarily to allogeneic stem cell transplant (“transplant indications”) or 15 diseases or conditions primarily under investigation for autologous stem cell infusion (“regenerative indications”), regardless of whether they planned to receive a transplant or infusion.

Results:  We received 43,259 completed responses.  Of the families completing the questionnaire, 1.87% reported at least one transplant indication and 17.04% reported at least one regenerative indication.  Figure 1 shows the total number and percent of families that reported each of the transplant and regenerative indications listed.  For transplant indications, after subtracting families where the child with the condition was the family member with stored cells, 1.68% of families reported at least one indication.  The most common indications reported were Hodgkin's lymphoma (0.33%), non-Hodgkin's lymphoma (0.32%), and acute lymphoblastic leukemia (0.25%) (Figure 2).  Similarly, for regenerative indications, after subtracting families where the child with the condition was not the family member with stored cells, 4.98% of families reported at least one indication.  The most common indications reported were autism/apraxia (1.99%), other developmental delay (1.41%), and childhood hearing loss (0.39%) (Figure 3).

Conclusion:  Among families who store newborn stem cells in private cord blood banks, conditions for which stem cell transplant or infusion may be indicated or under investigation are relatively prevalent, especially for regenerative indications.