Odds of Using Cord Blood

Traditional Stem cell Transplants

Cord blood is an accepted source of stem cells for any of the diseases where stem cell transplants are a standard therapy. This covers a long list of oncology and hematology diagnoses. Cord blood transplants have been in use since 1988, and by the end of 2009 there had been about 20,000 cord blood transplants world-wide (1). Yet, it was not until late 2011 that the US FDA finally licensed cord blood transplants for most of the diseases on the standard therapy list. While the FDA licensure does not change clinical practices at transplant centers, it does open up access to cord blood transplants for those patients whose health insurance providers had previously used the lack of licensure as a reason not to cover the transplants.

The definitive statistics on the rate of stem cell transplantation in the US were published in 2008 by Nietfeld et al. (2). The cumulative probability that a person will have some type of stem cell transplant by age 70, either from their own cells or from a donor, is approximately 1 in 200. This is not the probabiliity of needing a transplant, it is the probabilty of actually having one, assuming that there is no difficulty finding a matching donor. The study was based on transplant rates between 2001-2003 and relied on the national databases of diagnoses from SEER and transplantation from CIBMTR. The important caveat about this study, illustrated in the figure in the sidebar, is that rates of cancer increase with age, and thus the vast majority of transplant patients are older adults.

When parents contemplate cord blood banking, they consider the odds that their baby or an immediate family member will need a stem cell transplant. The cumulative probability that a child will have a stem cell transplant by age 10 is only 1 in 5,000 (.02%) for transplants from a donor and 1 in 10,000 (.01%) for transplants of the child's own cells. Medical societies have warned parents that the odds of their child needing stem cells from cord blood are very low, and if the only consideration is using the cord blood for a stem cell transplant, that is certainly true for the average person.


Diagnosis Transplant Type Stage of Use Odds by age 10
blood disease or
hereditary disorder
stem cell transplant from donor standard therapy 1 in 5,000
solid tumor transplant of your own stem cells standard therapy 1 in 10,000



Emerging Cell Therapies


Emerging cell therapies is a catch-all phrase for any new applications of cord blood stem cells that are currently in clinical trials. Success stories from these trials have received a great deal of media attention. But not every patient is a success story, and until follow up studies against controls are completed, it is not clear what fraction of the patients benefited from the therapy and the measure of benefit they received.

Parents usually wish to bank cord blood for their family on the premise that many more uses for the cord blood are around the corner, and that is probably true, but it is not a certainty. For this reason, family cord blood banking is a form of "health insurance", where parents make an investment based on what is known now, as a hedge against future developments.

There are a few pediatric disorders, summarized in Table 1 below, for which children are currently being treated with their own cord blood in clinical trials. What is most notable about these emerging therapies is that they all treat conditions that are not rare, conditions that a child is much more likely to experience than a stem cell transplant. The second important difference is that, whereas stem cell transplants require large cord blood collections, the emerging therapies can be successful with small collections.

The most well-known emerging therapy is to give a child his/her own cord blood stem cells for Cerebral Palsy. Since 2005, several hundred children world-wide have received this treatment for Cerebral Palsy and similar acquired neurological disorders (3). No one wants to imagine that their baby will be born with Cerebral Palsy, but the CDC estimates that 1 in 300 children in US elementary schools have Cerebral Palsy. The incidence of Cerebral Palsy among full term births is 0.2%, but among premature births the incidence is 10 times higher at 2% (4). For parents who are going through a pregnancy with a risk of premature birth, it is prudent to consider family cord blood banking in case the baby has this type of condition.

Lastly, there are currently (Feb. 2012) dozens of cell therapies in advanced (phase 3) clinical trials around the world that are using stem cells from bone marrow to treat medical conditions that afflict much of mankind: Stroke, heart disease, auto-immune disorders, neuro-degenerative disorders, and arthritis (ref: ClinicalTrials.gov). Just like cord blood can be substituted for bone marrow in stem cell transplants, any of the emerging therapies that are using stem cells from bone marrow could, in principle, use stem cells from cord blood or cord tissue. Table 2 below is a list of trials that are already using donor cord blood in cell thrapy for adult diseases.

Table 1. Emerging Therapies where children use their own Cord Blood Stem Cells


Diagnosis Occurence in USA Cell Therapy Stage of Use Clinical trial
Acquired Hearing Loss 12.5% ages 6-19 child's own cord blood clinical trial:
phase 1
NCT01343394
NCT02038972
Cerebral Palsy 0.2% full term births,
2.2% premature births,
1 in 300 kids ages 5-10
child's own cord blood clinical trials:
phase 2
NCT01147653
NCT01072370
NCT01988584
pending
Neonatal Oxygen Deprivation 0.2% full term births child's own cord blood clinical trials:
phase 1
NCT00593242
NCT01506258
Autism 1.13% (1 in 88) children child's own cord blood clinical trial:
phase 2
NCT01638819
Traumatic Brain Injury 435,000 per year ages 0-14, #1 cause of death in kids child's own cord blood clinical trial:
phase 1
NCT01251003
NCT01700166
Type 1 Diabetes 1.7 per 1000 ages birth-19 child's own cord blood clinical trials:
phase 1 & 2
NCT00989547
NCT00873925
CoRD
Hypoplastic Left Heart Syndrome 0.2 per 1000 births child's own cord blood clinical trial:
phase 1
NCT01445041
NCT01856049
Table copyright Frances Verter, PhD 2014 - References Below

Information graphic about stem cells and brain injury


Use of this graphic does not constitute an endorsement by Parent's Guide to Cord Blood Foundation


Table 2. Emerging Therapies using donated Cord Blood Stem Cells


Diagnosis Occurence in USA Cell Therapy Stage of Use Clinical trial
Cerebral Palsy 2 per 1000 full term births donor cord blood clinical trial:
phase 2
NCT01193660
NCT01528436
NCT01639404
NCT01991145
NCT02025972
premature lungs (BPD) 25% births under 1500gm donor cord blood clinical trial:
phase 2
NCT01897987
Type 1 Diabetes 1.7 per 1000 ages birth-19 donor cord blood clinical trial:
phase 2
NCT01350219
NCT01996228
cartilage repair 10-25% adolescents have knee injuries donor cord blood Cartistem approved by Korean FDA NCT01733186
Critical Limb Ischemia 2.5 per 1000 people, >80% of them diabetics donor cord blood clinical trial:
phase 1
NCT01019681
Table copyright Frances Verter, PhD 2014 - References Below


References for body of page

  1. Rocha, V & E Gluckman, Brit. J. Haematology 2009;147:262-274
  2. Nietfeld, JJ et al., Biol. Blood and Marrow Trans. 2008;14:316-322 (full text)
  3. Verter, F & JJ Nietfeld, Cytotherapy 2010;12:Suppl.1, abstract#157
  4. van Haastert, IC et al., Pediatrics 2011;159(1)86-91

References for tables of emerging therapies

Hearing Loss (trial is for acquired sensorineural type)
Statistics on 1 per 1000 births with bilateral sensorineural hearing loss of at least 40 dB from
Smith, RJH, Bale Jr, JF, & White, KR. Lancet 2005; 365:879-90 doi:10.1016/S0140-6736(05)71047-3
Statistic: 12.5% of children ages 6-19 (5.2 million) suffer noise-induced hearing loss. Niskar, AS, et al. JAMA.1998;279:1071-1075. AAP publications
Animal study: Revoltella RP, et al. Cell Transplant 2008;17(6):665-678 PMID:18819255
Cerebral Palsy
Statistic 2 per 1000 full term births is incidence rate from Centers for Disease Control (CDC)
Statistic 2.2% of premature births from van Haastert, I.C. et al. Pediatrics 2011; 159(1):86-91 doi:10.1016/j.jpeds.2010.12.053
Statistic 1 in 300 children ages 5-10 is prevalence from CDC.
Sun, JJ et al. Transfusion Sept. 2010; 50(9):1980-1987 doi:10.1111/j.1537-2995.2010.02720.x
Papadopoulos, K et al. Restorative Neurology and Neuroscience 2011; 29:17-22 doi:10.3233/RNN 20110572
Purandare, C et al. 2012; Case Reports in Transplantation (2012), Article ID825289, 6 pages doi:10.1155/2012/825289
Neonatal Oxygen Deprivation
Statistics Hypoxic Ischemic Encephalopathy (HIE) 2 per 1000 are from Smith, J. etal. BJOG 2000;107(4):4616 doi: 10.1111/j.1471-0528.2000.tb13262.x
Liao, Y, Cotten, M, Tan, S, Kurtzberg, J and MS Cairo, MS 2013; Bone Marrow Transplantation 48:890-900 doi:10.1038/bmt.2012.169
Premature Lungs
Statistics BronchoPulmonary Dysplasia (BPD) 25% babies born wieghing under 1500gm from Van Marter LJ. Semin Fetal Neonatal Med. 2009;14(6):358-66 doi: 10.1016/j.siny.2009.08.007.
Autism
Statistic 1 in 88 children are on the spectrum of autism related disorders is prevalence from CDC.
Traumatic Brain Injury
Statistics on occurence from Brain Injury Assoc. of America
Cox Jr., CS et al. Neurosurgery 2011; 68(3):588-600 doi: 10.1227/NEU.0b013e318207734c
Type 1 Diabetes
Statistic 1.7 per 1000 ages birth-19 is prevalence from Centers for Disease Control (CDC)
Haller MJ, et al. Diabetes Care 2011;34(12):2567-9. PMCID: PMC3220832
Yong Zhao, et al. BioMed Central Medicine 2012; 10:3 doi:10.1186/1741-7015-10-3
Science Daily Report on diabetes reversal with the "Stem Cell Educator" containing allogeneic cord blood.
Hypoplastic Left Heart Syndrome
Statistics 0.16-0.36 per 1000 live births from Fyler DC. Pediatrics 1980;65(2Pt2):375-461 (not on-line)
Cartilage Repair
Statistics on occurance from Louw et al. Brit. J Sports Med 2008;42(1)2-10 doi:10.1136/bjsm.2007.035360
News report on Cartistem approval
Critical Limb Ischemia
Statistics on occurance from Bitar, FG & Garcia, LA Vascular Disease Management 2010;7:E182-E184