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Literature / Reference

During pregnancy, if the mother suffers from organ damage, the baby in the womb sends stem cells to repair the damaged organ
Dawe GS, Tan XW, Xiao Z-C. Cell Migration from Baby to Mother. Cell Adhesion & Migration. 2007;1(1):19-27.
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2633676/ Accessed July 26, 2017

The probability of finding a donor stem cell source through a public bank is low. Banking cord blood with a family bank increases the chances of having a matched unit available.

Beatty PG, Boucher KM, Mori M, Milford EL. The probability of finding HLA-mismatched related or unrelated marrow or cord blood donors. Hum Immunol. 2000;61(8):834-840.

Graft versus Host Disease (GVHD) is a common side effect of any transplant when the immune cells of the recipient’s body do not recognize the donor’s cells and start attacking them, thus leading to failure of the transplant.

Lee, Stephanie J.; Vogelsang, Georgia; Flowers, Mary E.D. (2003). “Chronic graft-versus-host disease”. Biology of Blood and Marrow Transplantation. 9 (4): 215–33 

GVHD is broadly categorized into two types, the acute and chronic where its symptoms can range from modest to serious.

www.lls.org/…/f-73147405621_AvrIWIiW_bloodmarrowstemcelltransplantation.pdf. Accessed September 7, 2017

 

Cord blood is currently used in the treatment of around 80 diseases,
Data on file.  Retrieved June 13, 2017 from ViaCord.com .PerkinElmer, Inc.  Most of the diseases on this list are inherited genetic diseases.  Typically, these treatments require a donor transplant, such as from a sibling.  If your child needs a donor, a family member is always the first-choice source.  Treatments for cancers like neuroblastoma can use a child’s own cord blood.  Although the potential use of newborn stem cells is expanding rapidly, the odds a family member without a defined risk will need to use their child’s stem cells are low.  Banking cord blood does not guarantee that treatment will be effective and only a treating physi7cian can determine when it can be used.

Umbilical Cord Blood: Information for Childbirth Educators; Renece Waller-Wise, The Journal of Perinatal Education, 20(1), 54–60; Cord Blood Registry | Parent’s Guide to Cord Blood, July 19 2017; Cord Blood and the FDA – Parent’s Guide to Cord Blood, March 11 , 2016

Transplant outcomes are better with related cord blood donor cells compared with unrelated cord blood or bone marrow donor cells.
Gluckman E, Rocha V, Boyer-Chammard A, et al. For the Eurocord Transplant Group and the European Blood and Marrow Transplantation Group. N Engl J Med. 1997;337:373-381.

Hematopoietic cell transplantation (HCT) of umbilical cord origin can benefit for malignant and nonmalignant diseases like Fanconi’s anemia, aplastic anemia, leukemias, bone marrow failure syndromes, inborn error of metabolism, congenital disorders and hemoglobinopathies

  • Pai S-Y, Logan B, Griffith LM, et al. Transplantation outcomes for severe combined immunodeficiency, 2000-2009. N Engl J Med. 2014;371: 434–46.
  • Boelens JJ, Aldenhoven M, Purtill D, et al. Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning. Blood. 2013;121: 3981–7.
  • Myers KC, Davies S. Hematopoietic stem cell transplantation for bone marrow failure syndromes in children. Biol Blood Marrow Transplant. 2009;15(3):279–92.
  • King A, Shenoy S. Evidence-based focused review of the status of hematopoietic stem cell transplantation as treatment of sickle cell disease and thalassemia. Blood. 2014;123(20):3089–4.

Diseases Treatable with Cord Blood Transplant
Blood Disorders

  1. Leukemia
    Damaris Christensen; Umbilical Cord Blood Offers Another Option for Leukemia Patients. Journal of the National Cancer Institute, Volume 97, Issue 4, 16 February 2005, Pages 253–254.
    Laughlin M, Eapen M, Rubinstein P, et al. Outcomes after transplantation of cord blood or bone marrow from unrelated donors in adults with leukaemia. New England Journal of Medicine. 2004; 351:2265-2275.
  2. Lymphomas
    Yuji, K., Miyakoshi, S., Kato, D., Miura, Y., Myojo, T., & Murashige, N. et al. (2005). Reduced-intensity unrelated cord blood transplantation for patients with advanced malignant lymphoma. Biology Of Blood And Marrow Transplantation, 11(4), 314-318.

Blood cell proliferation disorders
1.Anaemias
Hassall, O. W., Thitiri, J., Fegan, G., Hamid, F., Mwarumba, S., Denje, D., … Bates, I. (2015). Safety and efficacy of allogeneic umbilical cord red blood cell transfusion for children with severe anaemia in a Kenyan hospital: an open-label single-arm trial. The Lancet. Haematology, 2(3), e101–e107.
Prasad, V., & Kurtzberg, J. (2009). Umbilical cord blood transplantation for non-malignant diseases. Bone Marrow Transplantation, 44(10), 643-651.

Inherited Immune System Disorders

  1. Severe Combined Immunodeficiency (SCID with Adenosine Deaminase Deficiency (ADA-SCID), SCID which is X-linked, SCID with absence of T & B Cells, SCID with absence of T Cells, Normal B Cells, Omenn Syndrome, Neutropenias, Infantile Genetic Agranulocytosis (Kostmann Syndrome), Myelokathexis)
    Dvorak, C., & Cowan, M. (2007). Hematopoietic stem cell transplantation for primary immunodeficiency disease. Bone Marrow Transplantation, 41(2), 119-126.
    Slatter, M., & Cant, A. (2011). Hematopoietic stem cell transplantation for primary immunodeficiency diseases. Annals Of The New York Academy Of Sciences, 1238(1), 122-131.
  2. Ataxia-Telangiectasia, Bare Lymphocyte Syndrome, DiGeorge Syndrome, Hemophagocytic Lymphohistiocytosis, Leukocyte Adhesion Deficiency, Leukocyte Adhesion Deficiency, Lymphoproliferative Disorder, X-linked (Susceptibility to Epstein-Barr virus), Wiskott-Aldrich Syndrome, Myeloproliferative Disorders, Acute Myelofibrosis, Agnogenic Myeloid Metaplasia (Myelofibrosis), Polycythemia Vera, Essential Thrombocythemia
    Knutsen, A., Steffen, M., Wassmer, K. and Wall, D. (2003). Umbilical cord blood transplantation in Wiskott Aldrich syndrome. The Journal of Pediatrics, 142(5), pp.519-523.
    Robin, M., Giannotti, F., Deconinck, E., Mohty, M., Michallet, M., Sanz, G., . . . Ruggeri, A. (2014). Unrelated Cord Blood Transplantation for Patients with Primary or Secondary Myelofibrosis. Biology of Blood and Marrow Transplantation, 20(11), 1841-1846.

Phagocyte Disorders

Chediak-Higashi Syndrome, Chronic Granulomatous Disease, Neutrophil Actin Deficiency, Reticular Dysgenesis
Bhattacharya, A., Slatter, M., Curtis, A., Chapman, C., Barge, D., Jackson, A., Flood, T., Abinun, M., Cant, A. and Gennery, A. (2003). Successful umbilical cord blood stem cell transplantation for chronic granulomatous disease. Bone Marrow Transplantation, 31(5), pp.403-405.
Reubsaet, L., Boelens, J., Rademaker, C., Smal, J. and Wulffraat, N. (2007). Successful cord blood transplantation in a premature and dysmature neonate of 1700 g with reticular dysgenesis. Bone Marrow Transplantation, 39(5), pp.307-308.
Zhang, Y., Gao, Z. and Yu, X. (2017). A case of Chediak-Higashi syndrome presented with accelerated phase could be treated effectively by unrelated cord blood transplantation. Pediatric Transplantation, p.e13014

Bone Marrow Cancers
Multiple Myeloma, Plasma Cell Leukemia, Waldenstrom’s Macroglobulinemi
Kawamura, K., Takamatsu, H., Ikeda, T., Komatsu, T., Aotsuka, N., Amano, I., Yamamoto, G., Watanabe, K., Ohno, Y., Matsue, K., Kouzai, Y., Tsukada, N., Ishiyama, K., Anzai, N., Kato, K., Suzuki, R., Sunami, K. and Kanda, Y. (2015). Cord Blood Transplantation for Multiple Myeloma: A Study from the Multiple Myeloma Working Group of the Japan Society for Hematopoietic Cell Transplantation. Biology of Blood and Marrow Transplantation, 21(7), pp.1291-1298.

Inherited Disorders of the Immune System & Other Organs
Cartilage-Hair Hypoplasia, Erythropoietic Porphyria, Hermansky-Pudlak Syndrome, Pearson’s Syndrome, Shwachman-Diamond Syndrome, Systemic Mastocytosis
Allewelt, H., Patel, S., Prasad, V., Kurtzberg, J., Driscoll, T., Martin, P., Page, K. and Parikh, S. (2016). Umbilical Cord Blood Transplantation for Cartilage Hair Hypoplasia: A Single Center Experience Demonstrates Excellent Outcomes Using Myeloablative Preparative Regimens. Biology of Blood and Marrow Transplantation, 22(3), p.S235.

Inherited Metabolic Disorders

  1. Hurler Syndrome (MPS-IH), Scheie Syndrome (MPS-IS), Hunter Syndrome (MPS-II), Sanfilippo Syndrome (MPS-III), Morquio Syndrome (MPS-IV), Maroteaux-Lamy Syndrome (MPS-VI), Sly Syndrome (MPS-VII) (beta-glucuronidase deficiency), Mucolipidosis II (I-cell Disease), Leukodystrophy Disorders, Adrenoleukodystrophy (ALD), Krabbe Disease (Globoid Cell Leukodystrophy), Metachromatic Leukodystrophy, Pelizaeus-Merzbacher Disease
    Mullen, C., Thompson, J., Richard, L. and Chan, K. (2000). Unrelated umbilical cord blood transplantation in infancy for mucopolysaccharidosis type IIB (Hunter syndrome) complicated by autoimmune hemolytic anemia. Bone Marrow Transplantation, 25(10), pp.1093-1097.
    Ooi, J. (2006). The efficacy of unrelated cord blood transplantation for adult myelodysplastic syndrome. Leukemia & Lymphoma, 47(4), pp.599-602.
    Prasad, V. K., Mendizabal, A., Parikh, S. H., Szabolcs, P., Driscoll, T. A., Page, K., … Kurtzberg, J. (2008). Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes. Blood, 112(7), 2979–2989.
    Stockman, J. (2006). Cord-Blood Transplants From Unrelated Donors in Patients With Hurler’s Syndrome. Yearbook of Pediatrics, 2006, pp.436-437.
    Wood, S., Allison, J., Escolar, M., Provenzale, J. and Kurtzberg, J. (2006). Neonatal umbilical cord blood transplantation favourably alters the neurodevelopmental outcome and survival of babies with infantile Krabbe disease. Biology of Blood and Marrow Transplantation, 12(2), pp.153-154.
  2. Niemann-Pick Disease, Sandhoff Disease, Wolman Disease
    Martin, P., Carter, S., Kernan, N., Sahdev, I., Wall, D., Pietryga, D., Wagner, J. and Kurtzberg, J. (2006). Results of the Cord Blood Transplantation Study (COBLT): Outcomes of Unrelated Donor Umbilical Cord Blood Transplantation in Pediatric Patients with Lysosomal and Peroxisomal Storage Diseases. Biology of Blood and Marrow Transplantation, 12(2), pp.184-194.
  3. Lesch-Nyhan Syndrome
    Kállay, K., Liptai, Z., Benyó, G., Kassa, C., Goda, V., Sinkó, J., Tóth, Á. and Kriván, G. (2012). Successful unrelated umbilical cord blood transplantation in Lesch-Nyhan syndrome. Metabolic Brain Disease, 27(2), pp.193-196.
  4. Osteoporosis/Osteopetrosis
    Chiesa, R., Ruggeri, A., Paviglianiti, A., Zecca, M., Gónzalez-Vicent, M., Bordon, V., Stein, J., Lawson, S., Dupont, S., Lanino, E., Abecasis, M., Al-Seraihy, A., Kenzey, C., Bierings, M., Locatelli, F., Gluckman, E., Schulz, A., Gennery, A., Page, K., Kurtzberg, J. and Rocha, V. (2016). Outcomes after Unrelated Umbilical Cord Blood Transplantation for Children with Osteopetrosis. Biology of Blood and Marrow Transplantation, 22(11), pp.1997-2002.`

Other References for Clinical Trials

CBR Clinical Trials and Stem Cell Research”. cordblood.com. CBR Systems, Inc. Retrieved 21 January 2014.

Cord Blood Registry | Parent’s Guide to Cord Blood, July 19 2017

Cord Blood and the FDA – Parent’s Guide to Cord Blood, March 11, 2016

National Cord Blood Program, 2015

National Marrow Donor Program 3 August 2011

Clinical Trials 3 August 2011

MedicineNet, 2012. Definition of Clinical Trials, 1 February 2014

Clinical trial registry page. ClinicalTrials.gov. Accessed Dec 2015.

Blood Sample from mother

http://www.nationalcordbloodprogram.org/work/collections.html Accessed July 25, 2017