Diseases Treated with HSC Stem Cells

Cord blood has an abundance of stem cells that can turn into all types of blood cells. These stem cells are known as Hematopoietic Stem Cells or HSCs. HSC stem cells are also found in everyone’s bone marrow.

HSC stem cells from cord blood, have been used to help people recovering from chemotherapy and radiation treatments for the last 29 years. The HSC treatment is known as Hematopoietic Stem Cell Transplants or HSCT as the stem cells are transplanted into the patient by way of an intravenous infusion to restore hematopoietic function.


Cord Blood Banking

Cord blood HSC stem cells can be retrieved from a public cord blood bank where the stem cells were donated for the common good of the community and can be used by anyone that is a close match (Allogeneic). HSC stem cells can also be stored in a private cord blood bank, but they can only be retrieved by the family that stored the cord blood stem cells (Autologous).

Acute graft-versus-host disease (GvHD) is a serious complication that affects about 35%-50% of allogeneic hematopoietic stem cell transplant (HSCT) recipients.[1] GvHD occurs when the donor’s T cells attack the recipient (host) tissues. GvHD complications can range from mild to fatal. GvHD can even occur in HLA identical siblings as the siblings will usually have genetically different proteins that would be considered foreign by the donor sibling’s T cells and possibly attacked.

HSCT treatments are currently being used for the following list of diseases.

Hodgkin Lymphoma[2]
Non-Hodgkin Lymphoma[3]
Waldenstrom’s Macroglobulinemia[4]

Aplastic Anemia[5]
Congenital Dyserythropoietic Anemia[6]
Diamond-Blackfan Anemia[7]
Fanconi Anemia – The first cord blood trasnplant was in 1988 from a full match sister.[8]
Paroxysmal Nocturnal Hemoglobinuria[9]
Pure Red Cell Aplasia[10]
Sickle Cell Disease[11]
Thalassemia Major[12]

Acute Lymphoblastic Leukemia (ALL)[13]
Chronic Lymphocytic Leukemia (CLL)[14]
Acute Myeloid Leukemia (AML)[15]
Chronic Myeloid Leukemia (CML)[16]
Biphenotype Acute Leukemia (BAL)[17]
Juvenile Myelomonocytic Leukemia (JMML)[18]
*JMML now includes diagnoses formerly referred to as chronic or JCML

Myelodysplastic Syndromes (MDS)
Refractory Cytopenia with Unilineage Dysplasia (RCUD)[19]
Refractory Anemia with Ringed Sideroblasts (RARS)[19]
Refractory Cytopenia with Multilineage Dysplasia (RCMD)[19]
Refractory Anemia with Excess Blasts-1 (RAEB-1)[19]
Refractory Anemia with Excess Blasts-2 (RAEB-2)[19]
Myelodysplastic Syndrome, Unclassified (MDS-U)[19]
Myelodysplastic Syndrome Associated with Isolated Del(5q)[19]
Chronic Myelomonocytic Leukemia (CMML)[19]

Mucopolysaccharidoses (MPS)
MPS I H Hurler a-L-Iduronidase[20]
MPS I S Scheie a-L-Iduronidase[20]
MPS I H-S Hurler-Scheie a-L-Iduronidase[20]
MPS VI Maroteaux-Lamy(arylsulfatase B) N-Acetylgalactosamine 4-sulfatase[21][22]
MPS VII Sly B-Glucuronidase[22]

Solid Tumors
Ewing Sarcoma[23]
Wilms’ Tumor[23]
Germ Cell Tumors[23]
Brain Tumors[23]

Platelet Disorders
Amegakaryocytic Thrombocytopenia[24]
Glanzmann Thrombasthenia[25]

Multisystem Disorders
Congenital Erythropoietic Porphyria (Gunther Disease)[26]
Shwachman Diamond Syndrome[27]
Advanced Systemic Mastocytosis[28]

Immunodeficiency Disorders
Omenn Syndrome[29]
SCID absence of T cells, absence of B cells (T–B- SCID)[30]
SCID absence of T cells, presence B cells (T–B+ SCID)[30]
SCID Adenosine Deaminase Deficiency (ADA-SCID)[30]
SCID X-linked (X-SCID)[30]
Common Variable Immunodeficiency[31]
DiGeorge Syndrome[32]
Hemophagocytic Lymphohistiocytosis[33]
Leukocyte Adhesion Deficiency[34]
Wiskott-Aldrich Syndrome[35]
Reticular Dysgenesis[36]
Cartilage Hair Hypoplasia[37]

Phagocytic Disorders
Chediak-Higashi Syndrome[38]
Chronic Granulomatous Disease[39]

Lysosomal Storage Diseases(LSD)[40]
Niemann-Pick Disease[41]
Wolman Disease[42]

Myeloproliferative Disorders
Essential Thrombocythemia[43]
Polycythemia Vera[43]

Neurodevelopmental Disorders
Lesch-Nyhan Syndrome[44]
Infantile Osteopetrosis[45]

X-linked Adrenoleukodystrophy (ALD)[46]
Krabbe Disease[47]
Metachromatic Leukodystrophy[48]

Bone Marrow Cancers
Multiple Myeloma(MM)[49]
Plasma Cell Leukaemia[50]


1. David J. et al. Orphanet J Rare Dis. 2007; 2: 35. PMCID:PMC2018687
2. Afonso José Pereira C. et al. Rev Bras Hematol Hemoter. 2011; 33(1): 10–14. PMCID:PMC3521428
3. Holmberg L. et al. Oncology (Williston Park). 2003 May;17(5):627-32, 635, 640; discussion 640-2. PMID:12800792
4. Anagnostopoulos A. et al. Biol Blood Marrow Transplant. 2006 Aug;12(8):845-54. PMID:16864055
5. Young M. et al. Curr Opin Hematol. 2013 Nov;20(6):515-20. PMID:24104412
6. Unal S. et al. Pediatr Transplant. 2014 Jun;18(4):E130-3. PMID:24724984
7. Mugishima H. Pediatr Transplant. 2007 Sep;11(6):601-7. PMID:17663681
8. Gluckman E et al. 2005; Cytotherapy. 2005;7(3):219-27. PMID:16081348
9. Robert A. Haematologica. 2010 Jun; 95(6): 855–856. PMCID: PMC2878778
10. Tseng S. et al. Am J Hematol. 2003 Dec;74(4):273-5. PMID:14635209
11. Monica B. et al. J Blood Med. 2015; 6: 229–238. PMCID:PMC4506029
12. Emanuele A. et al. Haematologica. 2014 May; 99(5): 811–820. PMCID:PMC4008115
13. Josep-Maria R. Haematologica. 2011 Aug; 96(8): 1083–1086. PMID:16081348
14. Douglas E. E et al. Curr Opin Oncol. 2012 Mar; 24(2): 176–181. PMCID:PMC4696047
15. Magenau J. et al. Curr Oncol Rep. 2013 Oct;15(5):436-44. PMID:23959811
16. Alok G. et al. Indian J Med Paediatr Oncol. 2014 Jul-Sep; 35(3): 207–210. PMCID:PMC4202616
17. Amal S. et al. Haematologica. 2009 Dec; 94(12): 1682–1690. PMCID:PMC2791935
18. Locatelli F. et al. Blood. 2005 Jan 1;105(1):410-9. Epub 2004 Sep 7. PMID:15353481
19. Mikkael A. et al. April 2014. Cleveland Clinic Foundation.
20. Lorne C. GeneReviews University of Washington, Seattle; 2016 Feb 11. PMID:20301341
21. Giugliani R. Pediatr Endocrinol Rev. 2014 Sep;12 Suppl 1:152-8. PMID:25345097
22. Peters C. Bone Marrow Transplantation (2003) 31, 229–239. doi:10.1038/sj.bmt.1703839
23. Hale G. Expert Rev Anticancer Ther. 2005 Oct;5(5):835-46. PMID:16221053
24. Fatma S. et al. Clin Med Insights Pathol. 2010; 3: 25–30. PMCID:PMC2999995
25. Wiegering V. et al. Klin Padiatr. 2011 May;223(3):173-5 PMID:21567370
26. Martinez P. et al. Pediatr Dermatol. 2013 Jul-Aug;30(4):484-9. PMID:23557135
27. Fleitz J. et al. Bone Marrow Transplant. 2002 Jan;29(1):75-9. PMID:11840149
28. Celalettin U. et al. Journal of Clinical Oncology 2014 32:29, 3264-3274
29. Mazzolari E. et al. Bone Marrow Transplant. 2005 Jul;36(2):107-14. PMID:15908971
30. Diana T. et al. Appl Clin Genet. 2012; 5: 67–80. PMCID:PMC3681194
31. Wehr C. et al. J Allergy Clin Immunol. 2015 Apr;135(4):988-97.e6. PMID:25595268
32. Amayiri N. et al. Pediatr Transplant. 2013 Jun;17(4):394-402. PMID:23692601
33. Simone C. et al. Haematologica November 2008 93: 1694-1701; Doi:10.3324/haematol.13142
34. Qasim W. et al. Pediatrics. 2009 Mar;123(3):836-40. PMID:19255011
35. Muñoz A. et al. Pediatr Hematol Oncol. 2007 Sep;24(6):393-402. PMID:17710656
36. Bertrand Y. et al. Bone Marrow Transplant. 2002 May;29(9):759-62. PMID:12040473
37. Bordon V. et al. Blood. 2010 Jul 8;116(1):27-35. PMID:20375313
38. Umeda K. et al. Pediatr Transplant. 2016 Mar;20(2):271-5. PMID:26511512
39. Seger R. Immunol Allergy Clin North Am. 2010 May;30(2):195-208. PMID:20493396
40. Lund T. Pediatr Endocrinol Rev. 2013 Nov;11 Suppl 1:91-8. PMID:24380127
41. Shah A. Pediatrics. 2005 Oct;116(4):1022-5. PMID:16199719
42. Tolar J. Bone Marrow Transplant. 2009 Jan;43(1):21-7. PMID:18776925
43. Lussana F. et al. Haematologica. 2014 May;99(5):916-21. PMID:24389309
44. Kállay K. et al. Metab Brain Dis. 2012 Jun;27(2):193-6. PMID:22350962
45. Orchard P. et al. Blood. 2015 Jul 9;126(2):270-6. PMID:26012570
46. Cartier N. et al. Brain Pathol. 2010 Jul;20(4):857-62. PMID:20626747
47. McGraw P. et al. Radiology. 2005 Jul;236(1):221-30. PMID:15987975
48. Boucher A. Orphanet J Rare Dis. 2015 Aug 7;10:94. PMID:26245762
49. Shah N. et al. Biol Blood Marrow Transplant. 2015 Jul;21(7):1155-66. PMID:25769794
50. Nonami A. et al. Jpn J Clin Oncol. 2007 Dec;37(12):969-72. PMID:18055567

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