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Odds of Using Banked Cord Blood
1. Overview
2. Types of Stem Cell Transplants versus Cord Blood Banks
3. Transplant Use To Date
4. Transplant Use Predicted
5. Potential Use for Emerging Therapies
6. References
2. Types of Stem Cell Transplants versus Cord Blood Banks
3. Transplant Use To Date
4. Transplant Use Predicted
5. Potential Use for Emerging Therapies
6. References
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1. Overview
When parents have to decide whether to bank cord blood, the decision
usually boils down to the question:
What are the odds that banked cord blood will ever be used?
This is a simple question which does not have a simple answer.
In the past, the only medical use for banked cord blood has been stem cell transplants. In the future, cord blood will probably be used for some regenerative medicine therapies in which the patient's own stem cells repair the body.
Even when considering strictly transplant use, there are two different pathways of possible use: There are two different types of stem cell transplants, autologous and allogeneic, and what type of bank you use to store cord blood determines both: for which type of transplant it may be used, and for which patients. Hence, in section 2. we explain the types of transplants and how they are connected to the types of banks.
Whereas this is a medical perspective on cord blood banking models, there is also a separate web page which explains the types of cord blood banks from an economic perspective.
In section 3. we review the past and current rates at which cord blood is being used for stem cell transplants, relying on information from cord blood banks regarding their inventory and transplant rates.
In section 4. we predict the lifetime probability that a person will have some type of stem cell transplant, based on the latest statistical study.
Finally, in section 5. we look beyond the current use of cord blood for stem cell transplants and review some of the emerging medical therapies for which cord blood may be used in the future. It is not possible to predict the rate at which cord blood might be used for these treatments, but we can give ballpark estimates of the number of people who are diagnosed with potentially treatable conditions.
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What are the odds that banked cord blood will ever be used?
This is a simple question which does not have a simple answer.
In the past, the only medical use for banked cord blood has been stem cell transplants. In the future, cord blood will probably be used for some regenerative medicine therapies in which the patient's own stem cells repair the body.
Even when considering strictly transplant use, there are two different pathways of possible use: There are two different types of stem cell transplants, autologous and allogeneic, and what type of bank you use to store cord blood determines both: for which type of transplant it may be used, and for which patients. Hence, in section 2. we explain the types of transplants and how they are connected to the types of banks.
Whereas this is a medical perspective on cord blood banking models, there is also a separate web page which explains the types of cord blood banks from an economic perspective.
In section 3. we review the past and current rates at which cord blood is being used for stem cell transplants, relying on information from cord blood banks regarding their inventory and transplant rates.
In section 4. we predict the lifetime probability that a person will have some type of stem cell transplant, based on the latest statistical study.
Finally, in section 5. we look beyond the current use of cord blood for stem cell transplants and review some of the emerging medical therapies for which cord blood may be used in the future. It is not possible to predict the rate at which cord blood might be used for these treatments, but we can give ballpark estimates of the number of people who are diagnosed with potentially treatable conditions.
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2. Types of Stem Cell Transplants versus Cord Blood Banks
There are two forms of stem cell transplants:
When you place cord blood into a bank, the type of cord blood bank determines the type of transplant for which the blood may be used.
(Aside: If your first child donated to a public bank and then your second child needs a transplant, then in theory you could get the first child's cord blood back for a related allogeneic transplant from a public bank. But this is very rare, in part because public banks discard at least half their donations, and also because there is no guarantee that the donation was not given to some one else.)
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- Autologous transplants - the patient uses his/her own stem cells
- Allogeneic transplants - the patient receives stem cells from another person, who is called the donor.
When you place cord blood into a bank, the type of cord blood bank determines the type of transplant for which the blood may be used.
- Public Banks - cord blood from anonymous donors goes to unrelated allogeneic transplants
- Private/Family Banks - the child whose cord blood is banked may use it for an autologous transplant OR it may be used by a close family member (usually a sibling) for a related allogeneic transplant.
(Aside: If your first child donated to a public bank and then your second child needs a transplant, then in theory you could get the first child's cord blood back for a related allogeneic transplant from a public bank. But this is very rare, in part because public banks discard at least half their donations, and also because there is no guarantee that the donation was not given to some one else.)
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3. Transplant Use To Date
Background from the National Marrow Donor Program (NMDP):
Public donations are a vital medical resource that saves the lives of patients right now.
The NMDP says that the cumulative total of all cord blood transplants from public banks is currently (summer 2008) 650 times the number from private banks. Plus, adult transplants often use up two cord blood units from the public inventory (Barker etal. 2005). Bear in mind that these are all allogeneic transplants using unrelated donors.
Private storage is medical insurance for the future needs of the family.
Since the probability of needing a stem cell transplant rises quickly with age (see section 4), it will be decades before most of the children whose cord blood is banked today require a transplant. Should that need arise, medical studies show that related allogeneic transplants (eg from a sibling) have better outcomes than unrelated allogeneic transplants (Gluckman etal. 1997).
The family banking business is also driven by emerging therapies (see section 5). Parents are banking on the ability to use stem cells to repair the body, called "regenerative medicine". The odds that a person will one day need to repair heart muscle or nerve cells are enormous compared with the odds of requiring a traditional stem cell transplant.
Trade-offs
The greatest irony of cord blood banking is that while most of the transplants come out of public banks, most of the inventory is stored in private banks. As of June 2008, the Association of Family Cord Blood Banks estimated that about 750,000 cord blood collections were stored in family banks in the United States. At the same time, the US public inventory resides primarily in the NMDP cord blood bank network (73,111 collections) and the NY Blood Center (36,638 collections), amounting to about 110,000 collections for public use. The reason for this disparity is economic: in private banks the client pays the processing fee, whereas most public banks are supported by government funding.
A brighter future?
In some other nations, such as Germany (see VITA34) and Hong Kong (see CryoLife), it is possible to combine public and private banking: parents who bank privately can have the HLA type of their child's cord blood listed on a public registry, and if it matches a transplant patient, they have the option to release it. The country Spain takes this one step further and mandates that parents must release the cord blood if it matches a transplant patient. In the United States, this is not possible. The primary obstacle is that US public banks are subject to regulatory standards which require that the collection staff and site be inspected, whereas in family banking the collection takes place wherever the child is delivered and is performed by the delivery staff. If the United States could someday adopt the models used in other nations, then parents could choose both public AND family banking, instead of public OR family banking.
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- The NMDP also operates a national network of cord blood banks. They work in partnership with an agency called CIBMTR
to maintain statistics on transplants throughout the United States.
- According to the NMDP, about 20% of US transplant patients, including both children and adults, currently (2008) receive cord blood transplants, and that fraction is steadily growing.
- World-wide,
there were 2000 cord blood transplants in the year 2006, and the NMDP
projects there will be 10,000 cord blood transplants per year world-wide by 2015.
- Public banking is a very international endeavor, because in 40% of transplants the donor child and the patient are located in different countries.
Public donations are a vital medical resource that saves the lives of patients right now.
The NMDP says that the cumulative total of all cord blood transplants from public banks is currently (summer 2008) 650 times the number from private banks. Plus, adult transplants often use up two cord blood units from the public inventory (Barker etal. 2005). Bear in mind that these are all allogeneic transplants using unrelated donors.
Private storage is medical insurance for the future needs of the family.
Since the probability of needing a stem cell transplant rises quickly with age (see section 4), it will be decades before most of the children whose cord blood is banked today require a transplant. Should that need arise, medical studies show that related allogeneic transplants (eg from a sibling) have better outcomes than unrelated allogeneic transplants (Gluckman etal. 1997).
The family banking business is also driven by emerging therapies (see section 5). Parents are banking on the ability to use stem cells to repair the body, called "regenerative medicine". The odds that a person will one day need to repair heart muscle or nerve cells are enormous compared with the odds of requiring a traditional stem cell transplant.
Trade-offs
The greatest irony of cord blood banking is that while most of the transplants come out of public banks, most of the inventory is stored in private banks. As of June 2008, the Association of Family Cord Blood Banks estimated that about 750,000 cord blood collections were stored in family banks in the United States. At the same time, the US public inventory resides primarily in the NMDP cord blood bank network (73,111 collections) and the NY Blood Center (36,638 collections), amounting to about 110,000 collections for public use. The reason for this disparity is economic: in private banks the client pays the processing fee, whereas most public banks are supported by government funding.
A brighter future?
In some other nations, such as Germany (see VITA34) and Hong Kong (see CryoLife), it is possible to combine public and private banking: parents who bank privately can have the HLA type of their child's cord blood listed on a public registry, and if it matches a transplant patient, they have the option to release it. The country Spain takes this one step further and mandates that parents must release the cord blood if it matches a transplant patient. In the United States, this is not possible. The primary obstacle is that US public banks are subject to regulatory standards which require that the collection staff and site be inspected, whereas in family banking the collection takes place wherever the child is delivered and is performed by the delivery staff. If the United States could someday adopt the models used in other nations, then parents could choose both public AND family banking, instead of public OR family banking.
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4. Transplant Use Predicted
The most accurate prediction to date of the probability that a person will have a stem cell transplant over the course of a lifetime was published in March 2008:
Nietfield J.J., Pasquini, M.C., Logan, B.R., Verter, F., and Horowitz, M.M.
Lifetime probabilities of hematopoietic stem cell transplantation in the U.S.
Biology of Blood and Marrow Transplantation. 2008;14:316-322
Downloads:
Nietfield J.J., Pasquini, M.C., Logan, B.R., Verter, F., and Horowitz, M.M.
Lifetime probabilities of hematopoietic stem cell transplantation in the U.S.
Biology of Blood and Marrow Transplantation. 2008;14:316-322
Downloads:
Cumulative Probability versus Age: (under current medical practices)
Probability of a person receiving an autologous transplant of her own cells is .02% by age 20, .05% by age 40, and .23% by age 70 years (ie: 1 in 435 by age 70).
Probability of a person receiving an allogeneic transplant of cells from a donor, with no limitations on donor availability, is .04% by age 20, .10% by age 40, and .25% by age 70 years (ie: 1 in 400 by age 70).
The net probability of a person receiving either type of transplant is .06% by age 20, .15% by age 40, and .46% by age 70 years (ie: 1 in 200 by age 70).
Probability of a person receiving an autologous transplant of her own cells is .02% by age 20, .05% by age 40, and .23% by age 70 years (ie: 1 in 435 by age 70).
Probability of a person receiving an allogeneic transplant of cells from a donor, with no limitations on donor availability, is .04% by age 20, .10% by age 40, and .25% by age 70 years (ie: 1 in 400 by age 70).
The net probability of a person receiving either type of transplant is .06% by age 20, .15% by age 40, and .46% by age 70 years (ie: 1 in 200 by age 70).
Explanatory notes for Nietfeld et al. 2008:
The paper is about "Hematopoietic" Stem Cell Transplants (aka HSCT), which basically means blood-forming stem cells like those found in bone marrow or cord blood.
Nietfeld et al. 2008 predicts the probability that a person in the US will receive a transplant, NOT that the person will be diagnosed with a disease or will be diagnosed and need a transplant.
The probabilities are based on national medical data bases (SEER and CIBMTR) for the years 2001-2003.
The only diseases included are those for which transplantation is a standard medical therapy, and also for which transplants amount to at least 5% of the total number of transplants (see Table 1 in the paper).
The only assumption added to the data is that patients who need a transplant from a donor will have one ("no donor limitations"). This goes into the allogeneic results and the net results.
The probability that a person will receive a stem cell transplant cannot be interpreted as the probability of receiving a cord blood transplant -- the selection of the best stem cell source for the circumstances will be made at the time of transplant.
Prior Publications:
All Medical Society Opinions about cord blood banking published prior to March 2008 are based on inaccurate statistics for the "odds of use".
Numerous publications have quoted the odds of use as between 1:10,000 and 1:200,000, when there is no original publication which calculates those numbers (Annas 1999, Greene & Cefalo 1999, Burgio, Gluckman, & Locatelli 2003, Ecker & Greene 2005).
There is only one prior paper which made a factual calculation of transplant probability:
Johnson calculated the probability of needing an autologous stem cell transplant by age 21. His result of 1 in 2700 or .04% is consistent with Nietfeld et al. 2008.
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The paper is about "Hematopoietic" Stem Cell Transplants (aka HSCT), which basically means blood-forming stem cells like those found in bone marrow or cord blood.
Nietfeld et al. 2008 predicts the probability that a person in the US will receive a transplant, NOT that the person will be diagnosed with a disease or will be diagnosed and need a transplant.
The probabilities are based on national medical data bases (SEER and CIBMTR) for the years 2001-2003.
The only diseases included are those for which transplantation is a standard medical therapy, and also for which transplants amount to at least 5% of the total number of transplants (see Table 1 in the paper).
The only assumption added to the data is that patients who need a transplant from a donor will have one ("no donor limitations"). This goes into the allogeneic results and the net results.
The probability that a person will receive a stem cell transplant cannot be interpreted as the probability of receiving a cord blood transplant -- the selection of the best stem cell source for the circumstances will be made at the time of transplant.
Prior Publications:
All Medical Society Opinions about cord blood banking published prior to March 2008 are based on inaccurate statistics for the "odds of use".
Numerous publications have quoted the odds of use as between 1:10,000 and 1:200,000, when there is no original publication which calculates those numbers (Annas 1999, Greene & Cefalo 1999, Burgio, Gluckman, & Locatelli 2003, Ecker & Greene 2005).
There is only one prior paper which made a factual calculation of transplant probability:
- Johnson, F.L., J Ped Hem Onc 1997; 19(3):183-186
- on-line version: Oct 1998 issue#43 of Blood & Marrow Transplant Newsletter (vol.9 no.3)
Johnson calculated the probability of needing an autologous stem cell transplant by age 21. His result of 1 in 2700 or .04% is consistent with Nietfeld et al. 2008.
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5. Potential Use for Emerging Therapies
This web site has a page on diseases treated by blood stem cells ("hematopoietic" stem cells), where the diseases are grouped into Standard Therapies, Therapies in Clinical Trials, and Experimental Treatments.
In this section we highlight two therapies in clinical trials which rely on autologous cord blood infusions to repair the body:
This web site has compiled a list of children who used their own cord blood. A chart compiled by CBR (see below), the world's largest cord blood bank, shows that in recent years the majority of cord blood released to their client families has been for autologous therapies.
In this section we highlight two therapies in clinical trials which rely on autologous cord blood infusions to repair the body:
- Type 1 Diabetes, also known as T1D or Juvenile Diabetes - A clinical trial at Shands Hospital, University of Florida, in Gainesville Florida USA, has shown promising results which were announced in a press release. Another clinical trial has been approved by the Diabetes Research Institute in Germany. The American Diabetes Association reports that 1 in 7000 children is diagnosed each year with Type 1 diabetes, and 1 in 600 children are living with it.
- Cerebral Palsy and other newborn brain injuries - Clinical trial at Duke University, in Durham, North Carolina, USA. There are anecdotal accounts of the results (Abby , Dallas) but no formal publication. The Brain Injury Association of America estimates that the prevalence of Cerebral Palsy is about 1 in 300 among children up to age 10.
This web site has compiled a list of children who used their own cord blood. A chart compiled by CBR (see below), the world's largest cord blood bank, shows that in recent years the majority of cord blood released to their client families has been for autologous therapies.
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6. References
Annas GJ.
Waste and longing--the legal status of placental-blood banking.
New England Journal of Medicine 1999; 340(19):1521-4.
Barker JN, Weisdorf DJ, DeFor TE, Blazar BR, McGlave PB, Miller JS, Verfaillie CM, Wagner JE.
Transplantation of 2 partially HLA-matched umbilical cord blood units to enhance engraftment in adults with hematologic malignancy.
Blood. 2005 Feb 1;105(3):1343-7.
Burgio GR, Gluckman E, Locatelli F.
Ethical reappraisal of 15 years of cord-blood transplantation.
Lancet. 2003; 361(9353):250-2.
Ecker JL, Greene MF.
The case against private umbilical cord blood banking.
Obstet Gynecol. 2005; 105(6):1282-4.
Gluckman, E., Rocha, V., Boyer-Chammard, A., Locatelli, F., Arcese, W., Pasquini, R., Ortega, J., Souillet, G.,
Ferreira, E., Laporte, J-P, Fernandez, M., and Chastang, C.
Outcome of Cord-Blood Transplantation from Related and UnRelated Donors
New England Journal of Medicine 1997; 337(6):373-381
Greene MF, Cefalo RC.
Obstetricians’ perspectives on umbilical cord blood storage.
Cancer Res Ther Control. 1999; 8:341-343.
Johnson, F.L.
Placental blood transplantation and autologous banking--caveat emptor.
J. Pediatric Hematol. Onc. 1997; 19(3):183-186
Nietfield J.J., Pasquini, M.C., Logan, B.R., Verter, F., and Horowitz, M.M.
Lifetime probabilities of hematopoietic stem cell transplantation in the U.S.
Biology of Blood and Marrow Transplantation. 2008; 14:316-322
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Waste and longing--the legal status of placental-blood banking.
New England Journal of Medicine 1999; 340(19):1521-4.
Barker JN, Weisdorf DJ, DeFor TE, Blazar BR, McGlave PB, Miller JS, Verfaillie CM, Wagner JE.
Transplantation of 2 partially HLA-matched umbilical cord blood units to enhance engraftment in adults with hematologic malignancy.
Blood. 2005 Feb 1;105(3):1343-7.
Burgio GR, Gluckman E, Locatelli F.
Ethical reappraisal of 15 years of cord-blood transplantation.
Lancet. 2003; 361(9353):250-2.
Ecker JL, Greene MF.
The case against private umbilical cord blood banking.
Obstet Gynecol. 2005; 105(6):1282-4.
Gluckman, E., Rocha, V., Boyer-Chammard, A., Locatelli, F., Arcese, W., Pasquini, R., Ortega, J., Souillet, G.,
Ferreira, E., Laporte, J-P, Fernandez, M., and Chastang, C.
Outcome of Cord-Blood Transplantation from Related and UnRelated Donors
New England Journal of Medicine 1997; 337(6):373-381
Greene MF, Cefalo RC.
Obstetricians’ perspectives on umbilical cord blood storage.
Cancer Res Ther Control. 1999; 8:341-343.
Johnson, F.L.
Placental blood transplantation and autologous banking--caveat emptor.
J. Pediatric Hematol. Onc. 1997; 19(3):183-186
Nietfield J.J., Pasquini, M.C., Logan, B.R., Verter, F., and Horowitz, M.M.
Lifetime probabilities of hematopoietic stem cell transplantation in the U.S.
Biology of Blood and Marrow Transplantation. 2008; 14:316-322
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Last modified: 21.January 2010
Copyright 2000 - 2010 Frances Verter
Copyright 2000 - 2010 Frances Verter