Comparison of children diagnosed with cerebral palsy in a private cord blood bank to an epidemiological sample.

BACKGROUND: Although cord blood (CB) stem cell research is being conducted for treatment of cerebral palsy (CP), little is known about children with CP and stored CB. AIMS: To compare demographic and clinical characteristics of children with CP and stored CB to children with CP identified in a population-based study. METHODS AND PROCEDURES: The Longitudinal Umbilical Stem cell monitoring and Treatment REsearch (LUSTRE®) Registry recruited children from the largest US private CB bank. Demographics, co-morbidities, and gross motor function (GMFCS level and walking ability) were collected and, where possible, compared with the CDC's Autism and Developmental Disabilities Monitoring (ADDM) Network. OUTCOMES AND RESULTS: 114 LUSTRE participants were compared to 451 ADDM participants. LUSTRE participants were more likely to be white, but sex distribution was similar. Co-morbidities (autism and epilepsy) and functional mobility were also similar. CONCLUSIONS AND IMPLICATIONS: The results of this analysis suggest that while children diagnosed with CP and with access to stored CB differ from a broader population sample in terms of demographics, they have similar clinical severity and comorbidity profiles. As such, LUSTRE may serve as a valuable source of data for the characterization of individuals with CP, including individuals who have or will receive CB infusions.

Prevalence of Medical Conditions Potentially Amenable to Cellular Therapy among Families Privately Storing Umbilical Cord Blood.

Introduction Little is known about the prevalence of conditions potentially amenable to cellular therapy among families storing umbilical cord blood in private cord blood banks. Methods A cross-sectional study of families with at least one child who stored umbilical cord blood in the largest private cord blood bank in the United States was performed. Respondent families completed a questionnaire to determine whether children with stored cord blood or a first-degree relative had one or more of 16 conditions amenable primarily to allogeneic stem cell transplant ("transplant indications") or 16 conditions under investigation for autologous stem cell infusion ("regenerative indications"), regardless of whether they received a transplant or infusion. Results 94,803 families responded, representing 33.3 % of those surveyed. Of respondent families, 16.01 % indicated at least one specified condition. 1.64 % reported at least one first-degree member with a transplant indication potentially treatable with an allogeneic stem cell transplant. The most common transplant indications reported among first-degree family members were Non-Hodgkin's Lymphoma (0.33 %), Hodgkin's Lymphoma (0.30 %), and Acute Lymphoblastic Leukemia (0.28 %). 4.23 % reported at least one child with a regenerative indication potentially treatable with an autologous stem cell infusion. The most common regenerative indications among children with stored umbilical cord blood were Autism/Autism Spectrum Disorder/Apraxia (1.93 %), Other Developmental Delay (1.36 %), and Congenital Heart Defect (0.87 %). Discussion Among families storing umbilical cord blood in private cord blood banks, conditions for which stem cell transplant or infusion may be indicated, or are under investigation, appear to be prevalent, especially for regenerative medicine indications.

The HI-STAR study: resource utilization and costs associated with serologic testing for antibody-positive patients at four United States medical centers.

BACKGROUND: Little is known about how the resource utilization and costs of serologic work ups for positive antibody screens vary across subpopulations based on diagnosis, transfusion history, and serologic testing history. STUDY DESIGN AND METHODS: Detailed data were collected on patient demographics, diagnoses, transfusion history, history of known allo- and autoantibodies, and specific serologic tests performed for 6077 consecutive serologic work ups in 3608 antibody-positive patients between 2009 and 2011 at four US academic medical centers. Direct testing costs were also determined at each site for each serologic test performed to calculate total costs per work up and per patient over the duration of the study. RESULTS: The mean direct cost of serologic testing was $114 per work up and $195 per patient. The mean cost per patient was significantly higher for 12 of 19 diagnostic categories evaluated, including autoimmune hemolytic anemia (mean cost per patient, $1490; p < 0.001), hematologic malignancies ($640, p < 0.001), and transplant recipients ($462, p = 0.019). Patient transfusion and serologic testing characteristics associated with greatest increases in costs included history of a warm autoantibody ($626, p < 0.001) and more than five prior transfusions ($404, p < 0.001). CONCLUSION: Antibody-positive patients with complex diagnoses or transfusion histories require significantly more resources and incur greater cost to assess red blood cell antibody status.