Prevalence of Medical Conditions Potentially Amena

Authors: Peter Mazonson Mark Kane Kelin Colberg Heather Harris Heather Brown Andrew Mohr Alyssa Ziman Chris Santas

Publish Date: 2016/08/16

Volume: 21, Issue: 1, Pages: 208-214

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Abstract

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 crosssectional 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 firstdegree 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 94803 families responded representing 333  of those surveyed Of respondent families 1601  indicated at least one specified condition 164  reported at least one firstdegree member with a transplant indication potentially treatable with an allogeneic stem cell transplant The most common transplant indications reported among firstdegree family members were NonHodgkin’s Lymphoma 033  Hodgkin’s Lymphoma 030  and Acute Lymphoblastic Leukemia 028  423  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 193  Other Developmental Delay 136  and Congenital Heart Defect 087  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 indicationsThe identification of umbilical cord blood UCB as a source of stem cells has made cord blood banking an important topic Yet among families with stored UCB the prevalence of conditions either known to be treatable or under investigation for treatment with UCB is largely unknown We believe this is the first study of disease prevalence in a private cord blood bank Among families surveyed 164  reported at least one firstdegree member with an indication potentially treatable with an allogeneic stem cell transplant while 423  reported at least one child with an indication under investigation for treatment with an autologous stem cell infusionUmbilical cord blood UCB is a rich source of hematopoietic stem cells Since the first successful sibling cord blood transplant for Fanconi anemia in 1988 UCB has been used to treat a variety of lifethreatening conditions including hematologic malignancies hemoglobinopathies and metabolic and immune disorders Gluckman et al 2011 More recently research has suggested that UCB has the potential to play a role in regenerative medicine applications where it may promote repair of organs and tissues outside of its hematopoietic lineage Harris et al 2007 Willert et al 2008 In these applications it is thought that UCB may repair damaged tissues either via cell differentiation and replacement or more likely through the release of antiinflammatory and other factors that stimulate endogenous repair mechanisms Willert et al 2008 Hau et al 2008 Neuhoff et al 2007 As a result new applications for UCB are now actively being investigated in the laboratory and in clinical trials Currently ten US based clinical trials are being conducted in pediatric populations to investigate the regenerative medicine potential of UCB Electronic Resource 1 A range of conditions are being studied including cerebral palsy hearing loss hypoplastic left heart syndrome hypoxicischemic encephalopathy pediatric stroke type I diabetes and autism “US National Institutes” 2015Traditionally in transplant medicine UCB from a healthy human leukocyte antigen HLAmatched donor is used in an allogeneic transplant to repopulate the recipient’s bone marrow after a preconditioning regimen In contrast in the United States the experimental use of UCB for regenerative medicine indications is often referred to as a stem cell “infusion” and almost always autologous Therefore although autologous transplants may be performed for traditional indications and allogeneic infusions may be performed for regenerative indications for the purpose of this study the term “transplant indications” refers exclusively to medical conditions for which UCB transplants are more likely to be done in an allogeneic fashion whereas the term “regenerative indications” refers exclusively to medical conditions for which UCB infusions are more be likely to be done in an autologous fashionUntil recently UCB was considered medical waste Forraz and McGuckin 2011 Badowski and Harris 2012 Soon after the first successful demonstration of UCB as a stem cell source cord blood banking was established in the United States As of 2013 it was conservatively estimated that at least 136 million cord blood units were banked in the United States “Parent’s Guide to Cord Blood” 2014 Families have the choice to either store cord blood in a private cord blood bank or to donate it to the public cord blood banking system Private and public cord blood banks are designed to serve different needs For a fee private banks reserve the cord blood unit exclusively for use by the donor and immediate family members while UCB donated to a public bank may be used by any patient in need though rarely the actual donor Private cord blood banking continues to grow According to a 2009 estimate private cord blood banks now store approximately 60  of the more than half million UCB units processed each year worldwide “World Stem Cell Summit” 2009Despite growing participation little information has been collected on families who choose to privately bank their children’s UCB This study was carried out to better characterize this population and to determine the prevalence of diseases and conditions potentially amenable to UCB transplants or infusions among families at a large private cord blood bankWe conducted a crosssectional survey using an online onepage Family Health Questionnaire FHQ of selfreported disease prevalence among all eligible families with at least one living child with stored UCB These families were identified using a contact database maintained by the largest private cord blood bank in the United States All families that elected to collect and store UCB from January 1994 to May 2014 were initially included Families were ineligible to receive an FHQ if they did not have a valid email address on file in the company database had a “donotcontact” request in place had a deceased child with stored UCB or had a deceased primary parent contact Additionally families with only adopted or surrogate births were not included due to the FHQ’s focus on biological relatives These exclusions accounted for approximately 17  of the active storage population

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