POLICY STATEMENT Organizational Principles to Guide and Define the Child Health Care System and/or Improve the Health of all Children William T. Shearer, MD, PhD, FAAP,a Bertram H. Lubin, MD, FAAP, b Mitchell S. Cairo, MD, FAAP, c CordLuigi D. Notarangelo, Blood MD, d SECTION ONBanking HEMATOLOGY/ONCOLOGY, SECTIONfor ON Potential ALLERGY AND IMMUNOLOGY Future Transplantation This policy statement is intended to provide information to guide abstract pediatricians, obstetricians, and other medical specialists and health care providers in responding to parents’ questions about cord blood donation and banking as well as the types (public versus private) and quality of cord blood banks. Cord blood is an excellent source of stem cells for hematopoietic stem cell transplantation in children with some aDepartment of Pediatrics and Pathology and Immunology, College of Medicine, Baylor University and Section of Immunology, Allergy, and fatal diseases. Cord blood transplantation offers another method of Rheumatology, Texas Children’s Hospital, Houston, Texas; bChildren’s Health, University of California, San Francisco and Benioff Children’s definitive therapy for infants, children, and adults with certain hematologic Hospital, San Francisco, California; cDepartment of Pediatrics, malignancies, hemoglobinopathies, severe forms of T-lymphocyte and other Medicine, Pathology, Microbiology and Immunology, and Cell Biology and Anatomy, Maria Fareri Children’s Hospital and New York Medical immunodeficiencies, and metabolic diseases. The development of universal College, Valhalla, New York; and dLaboratory of Host Defenses, National screening for severe immunodeficiency assay in a growing number of states Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland is likely to increase the number of cord blood transplants. Both public and All of the authors shared responsibility for writing and editing the private cord blood banks worldwide hold hundreds of thousands of cord document and reviewing and responding to questions and comments blood units designated for the treatment of fatal or debilitating illnesses. The from reviewers and the Board of Directors. procurement, characterization, and cryopreservation of cord blood is free This document is copyrighted and is property of the American Academy of Pediatrics and its Board of Directors. All authors have for families who choose public banking. However, the family cost for private filed conflict of interest statements with the American Academy banking is significant and not covered by insurance, and the unit may never of Pediatrics. Any conflicts have been resolved through a process approved by the Board of Directors. The American Academy of be used. Quality-assessment reviews by several national and international Pediatrics has neither solicited nor accepted any commercial accrediting bodies show private cord blood banks to be underused for involvement in the development of the content of this publication. treatment, less regulated for quality control, and more expensive for the Policy statements from the American Academy of Pediatrics benefit from expertise and resources of liaisons and internal (AAP) and family than public cord blood banks. There is an unquestionable need to external reviewers. However, policy statements from the American Academy of Pediatrics may not reflect the views of the liaisons or the study the use of cord blood banking to make new and important alternative organizations or government agencies that they represent. means of reconstituting the hematopoietic blood system in patients The guidance in this statement does not indicate an exclusive course with malignancies and blood disorders and possibly regenerating tissue of treatment or serve as a standard of medical care. Variations, taking systems in the future. Recommendations regarding appropriate ethical into account individual circumstances, may be appropriate. and operational standards (including informed consent policies, financial All policy statements from the American Academy of Pediatrics automatically expire 5 years after publication unless reaffirmed, disclosures, and conflict-of-interest policies) are provided for physicians, revised, or retired at or before that time. institutions, and organizations that operate or have a relationship with DOI: https:// doi. org/ 10. 1542/ peds. 2017- 2695 cord blood banking programs. The information on all aspects of cord blood banking gathered in this policy statement will facilitate parental choice for To cite: Shearer WT, Lubin BH, Cairo MS, et alAAP SECTION public or private cord blood banking. ON HEMATOLOGY/ONCOLOGY, AAP SECTION ON ALLERGY AND IMMUNOLOGY. Cord Blood Banking for Potential Future Transplantation. Pediatrics. 2017;140(5):e20172695 Downloaded from www.aappublications.org/news by guest on September 29, 2021 PEDIATRICS Volume 140, number 5, November 2017:e20172695 FROM THE AMERICAN ACADEMY OF PEDIATRICS INTRODUCTION TABLE 1 Diseases Treated by Umbilical Cord Blood Transplant by Using Either of hematopoietic stem cells to meet Sibling or Unrelated Donors the increased use of cord blood in Malignant Diseases Nonmalignant Diseases Hematopoietic stem cells have the older children and adults, as was Acute lymphocytic Fanconi anemia potential to reconstitute any blood seen in 1 limited– study (which leukemia cell type and rescue children with requires3, 15 confirmation17 in a larger Acute myelocytic Idiopathic aplastic malignancies and other types of life- study). The implementation leukemia anemia threatening diseases. The need for of universal newborn screening by Juvenile chronic Thalassemia myelogenous cord blood transplantation to treat using dried blood on filter paper and leukemia malignancies, hemoglobinopathies, the T-lymphocyte receptor excision Chronic Sickle cell anemia primary immunodeficiencies, and circle assay in more than 3000000 myelogeneous leukemia metabolic diseases1 within the newborn infants permitted the last decade has prompted the recognition of the true incidence of Neuroblastoma Amegakaryocytic thrombocytopenia American Academy of Pediatrics SCID requiring HSCT; it was found to “ Myelodysplastic Kostman syndrome (AAP) to revise its 2007 policy be nearly double (ie, 1 in 58000 live ” 18 syndrome statement Cord Blood Banking for2 births) previous estimates. This Blackfan-Diamond Potential Future Transplantation. new development has prompted the syndrome This revised policy statement search for a more convenient method Severe combined immunodeficiency of HSCT, a method that applies to the reviews the new applications for X-linked transplantation of the hematopoietic use of cord blood. However, other lymphoproliferative cells contained in cord blood sources of hematopoietic stem cells syndrome taken from the placenta of normal (eg, haploidentical parent donors) Wiskott-Aldrich newborn infants, blood that was may prove useful for patients syndrome 19, 20 Hurler syndrome previously discarded because of a with infections. Perhaps the 3 Hunter syndrome lack of need for it. By 2013, more most immediate challenge is that Gunther disease than 30 000 hematopoietic stem of educating medical personnel, Osteopetrosis cell transplants (HSCTs) have been parents, and the public about the Globoid cell performed by using cord blood as increasing need and uses of cord leukodystrophy 4, 5 Adrenoleukodystrophy blood banking. the source of stem cells worldwide. Lesch-Nyhan syndrome The disease distributions were In the following sections, several Adapted from Cairo MS, Rocha V, Gluckman E, Hale 57% for malignancies, 32.5% for important features of public G, Wagner J. Alternative allogenic donor sources for hemoglobinopathies, 6% for severe versus private cord blood banking transplantation for childhood diseases: unrelated cord combined immunodeficiency disease blood and haploidentical family donors [published are compared: (1) purpose; (2) correction appears in Biol Blood Marrow Transplant. (SCID) or related T-lymphocyte quality control and accreditation; 2008;14(11):1317–1318]. Biol Blood Marrow Transplant. 2008;14(1, suppl 1):44 53. Copyright 2008 American disorders,6, and 7 1.5% for other (3) cost and use of banked cord – © Society for Blood and Marrow Transplantation. disorders (Table 1). Approximately blood; (4) education of medical 800000 cord blood units are being personnel, expectant parents, and stored in public cord blood banks, the public; (5) summary; and (6) and more than 5000000 cord ’ recommendations. A review of these need to be aware that should their blood units are stored in private 8 components will enable parents and child develop leukemia, the child s cord blood banks. The trajectory medical personnel to make informed of increase in the number of cord stored cord blood already contains decisions on public or private cord premalignant cells and cannot, blood units being transplanted in blood banking for patients. patients older than 16 years has therefore, be used to treat21, the22 child PUBLIC VERSUS PRIVATE CORD BLOOD (autologous transplant). In that progressively increased over the past BANKING: PURPOSE AND OPINIONS decade (Fig 1). Clinical outcomes of case, the cord blood from an unrelated both malignant and nonmalignant Purpose child (an allogeneic transplant) disorders are beginning to emerge is needed. When there is a known in the literature, and it is certain that genetic defect in a family, there can HLA barriers can be breached more The primary goal of this policy be a need for directed