Skip to main content

Stem Cell Transplantation in Pediatrics

  • Chapter
  • First Online:
Pediatric Hematopoietic Stem Cell Transplantation for Pharmacists

Abstract

The concepts of cellular biology and the classification of stem cells were described in the previous chapter. In this chapter, the focus will be only on hematopoietic stem cells, which we can use today for stem cell transplantation in pediatric oncological/hematological patients. Hematopoietic stem cells are the most thoroughly characterized tissue-specific stem cells and have been experimentally studied for more than 50 years. The transplantation we work with in pediatric oncology/hematology area uses multipotent hematopoietic stem cells; they can generate all functional hematopoietic lineages in blood, including erythrocytes, leukocytes, and platelets; therefore, its transplantation solves the inappropriate functioning of the hematopoietic system in diseases such as leukemia and anemia. Hematopoietic stem cells for transplantation are collected from the bone marrow, peripheral blood, or umbilical cord blood. The transplantation can be autologous, with stem cells from the patient, or allogenic, with stem cells from a donor. When the donor is an identical twin, the transplantation is named syngeneic [51].

This is a preview of subscription content, log in via an institution to check access.

Access this chapter

Chapter
USD 29.95
Price excludes VAT (USA)
  • Available as PDF
  • Read on any device
  • Instant download
  • Own it forever
eBook
USD 79.99
Price excludes VAT (USA)
  • Available as EPUB and PDF
  • Read on any device
  • Instant download
  • Own it forever
Softcover Book
USD 99.99
Price excludes VAT (USA)
  • Compact, lightweight edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info

Tax calculation will be finalised at checkout

Purchases are for personal use only

Institutional subscriptions

References

  1. Zakrzewski W, Dobrzyński M, Szymonowicz M, Rybak Z. Stem cells: past, present, and future. Stem Cell Res Ther. 2019;10(1):1–22. https://doi.org/10.1186/s13287-019-1165-5.

    Article  CAS  Google Scholar 

  2. Hastings CA, Torkildson JC, Agrawal AK. Handbook of pediatric hematology and oncology: children’s Hospital & Research Center Oakland. 2nd ed. New York: Wiley Blackwell; 2013.

    Google Scholar 

  3. Dessels C, Alessandrini M, Pepper MS. Factors influencing the umbilical cord blood stem cell industry: an evolving treatment landscape. Stem Cells Transl Med. 2018;7(9):643–50. https://stemcellsjournals.onlinelibrary.wiley.com/doi/full/10.1002/sctm.17-0244 (Aug 13, 2019).

    Article  Google Scholar 

  4. Holtick U, Albrecht M, Chemnitz JM, et al. Bone marrow versus peripheral blood allogeneic haematopoietic stem cell transplantation for haematological malignancies in adults. Cochrane Database Syst Rev. 2014;(4):CD010189. https://doi.org/10.1002/14651858.CD010189.pub2.

  5. O’Donnell PV, Eapen M, Horowitz MM, et al. Comparable outcomes with marrow or peripheral blood as stem cell sources for hematopoietic cell transplantation from haploidentical donors after non-ablative conditioning: a matched-pair analysis. Bone Marrow Transplant. 2016;51:1599–601.

    Article  Google Scholar 

  6. Lapidot T, Dar A, Kollet O. How do stem cells find their way home? Blood. 2005;106:1901–10.

    Article  CAS  Google Scholar 

  7. Taketomo CK, Hodding JH, Kraus DM. Pediatric & neonatal dosage handbook. 21st ed. Hudson: Lexicomp; 2014.

    Google Scholar 

  8. McCune J, Jacobson P, Wiseman A, Militano O. Optimizing drug therapy in pediatric stem cell transplantation: focus on pharmacokinetics. Bone Marrow Transplant. 2015;50(2):165–72.

    Article  CAS  Google Scholar 

  9. Pulsipher MA, Boucher KM, Wall D, et al. Reduced-intensity allogeneic transplantation in pediatric patients ineligible for myeloablative therapy: results of the Pediatric Blood and Marrow Transplant Consortium Study ONC0313. Blood. 2009;114(7):1429–36.

    Article  CAS  Google Scholar 

  10. Atilla E, Atilla PA, Demirer T. A review of myeloablative vs reduced intensity/non-myeloablative regimens in allogeneic hematopoietic stem cell transplantations. Balkan Med J. 2017;34(1):1–9.

    Article  Google Scholar 

  11. Vettenranta K. Current European practice in pediatric myeloablative conditioning. Bone Marrow Transplant. 2008;41:S14–7.

    Article  Google Scholar 

  12. Socié G, Blazar BR. Overview of the immune biology of allogeneic hematopoietic stem cell transplantation. In: Immune biology of allogeneic hematopoietic stem cell transplantation. London: Elsevier Academic Press; 2012.

    Google Scholar 

  13. Falkenburg JHF, Jedema I. Graft versus tumor effects and why people relapse. Hematology Am Soc Hematol Educ Program. 2017;1:693–8.

    Article  Google Scholar 

  14. National Cancer Institute. www.cancer.gov (July 4, 2019).

  15. Hayani A, Lampeter E, Viswanatha D, Morgan D, Salvi SN. First report of autologous cord blood transplantation in the treatment of a child with leukemia. Pediatrics. 2007;119(1):e296–300. https://pediatrics.aappublications.org/content/119/1/e296 (Aug 12, 2019).

    Article  Google Scholar 

  16. Oliansky DM, Camitta B, Gaynon P, et al. Role of cytotoxic therapy with hematopoietic stem cell transplantation in the treatment of pediatric acute lymphoblastic leukemia: update of the 2005 evidence-based review. Biol Blood Marrow Transplant. 2012;18(4):505–22.

    Article  CAS  Google Scholar 

  17. Yeşilipek MA. Hematopoietic stem cell transplantation in children. Turk Pediatri Ars. 2014;49(2):91–8.

    Article  Google Scholar 

  18. Giebel S, Labopin M, Potter M, et al. Comparable results of autologous and allogeneic haematopoietic stem cell transplantation for adults with Philadelphia-positive acute lymphoblastic leukaemia in first complete molecular remission: an analysis by the Acute Leukemia Working Party of the EBMT. Eur J Cancer. 2018;96:73–81.

    Article  Google Scholar 

  19. Im HJ. Current treatment for pediatric acute myeloid leukemia. Blood Res. 2018;53(1):1–2.

    Article  Google Scholar 

  20. Brooke RJ, Bhatt NS, Li Y, et al. Reduced intensity vs myeloablative conditioning regimen for pediatric therapy-related myelodysplastic syndrome/acute myeloid leukemia. Biol Blood Marrow Transplant. 2019;25:S7–S75.

    Google Scholar 

  21. Khandelwal P, Millard HR, Thiel E, et al. Hematopoietic stem cell transplant activity in pediatric cancer between 2008 and 2014 in the United States: a CIBMTR report. Biol Blood Marrow Transplant. 2018;23(8):1342–9.

    Article  Google Scholar 

  22. Giulino-Roth L, Ricafort R, Kernan NA, et al. Ten-year follow-up of pediatric patients with non-Hodgkin lymphoma treated with allogeneic or autologous stem cell transplantation. Pediatr Blood Cancer. 2013;60(12):2018–24.

    Article  Google Scholar 

  23. Moskowitz CH, Walewski J, Nademanee A, et al. Five-year PFS from the AETHERA trial of brentuximab vedotin for Hodgkin lymphoma at high risk of progression or relapse. Blood. 2018;132(25):2639–42.

    Article  CAS  Google Scholar 

  24. Ishizawa K, Yanai T. Hematopoietic stem cell transplantation and brentuximab vedotin for patients with relapsed or refractory Hodgkin lymphoma and systemic anaplastic large-cell lymphoma. Adv Ther. 2019;36:2679.

    Article  CAS  Google Scholar 

  25. Locatelli F, Mauz-Koerholz C, Neville K, et al. Brentuximab vedotin for paediatric relapsed or refractory Hodgkin’s lymphoma and anaplastic large-cell lymphoma: a multicentre, open-label, phase 1/2 study. Lancet Haematol. 2018;5(10):e450–61.

    Article  Google Scholar 

  26. Styczyński J, Dębski R, Krenska A, Czyżewski K, Wysocki M. High-dose therapy and autologous hematopoietic cell transplantation rescue in children with neuroblastoma and Ewing sarcoma. Med Biol Sci. 2016;30(4):71–5.

    Article  Google Scholar 

  27. Drabko K, Raciborska A, Bilska K, et al. Consolidation of first-line therapy with busulphan and melphalan, and autologous stem cell rescue in children with Ewing’s sarcoma. Bone Marrow Transplant. 2012;47:1530–4.

    Article  CAS  Google Scholar 

  28. Proust-Houdemont S, Pasqualini C, Blanchard P, et al. Busulfan-melphalan in high-risk neuroblastoma: the 30-year experience of a single institution. Bone Marrow Transplant. 2016;51:1076–81.

    Article  CAS  Google Scholar 

  29. Pahnke S, Egeland T, Halter J, et al. Current use of biosimilar G-CSF for haematopoietic stem cell mobilisation. Bone Marrow Transplant. 2019;54:858–66.

    Article  Google Scholar 

  30. Gilman AL, Jacobsen C, Bunin N, et al. Phase I study of tandem high-dose chemotherapy with autologous peripheral blood stem cell rescue for children with recurrent brain tumors: a pediatric blood and marrow transplant consortium study. Pediatr Blood Cancer. 2011;57:506–13.

    Article  Google Scholar 

  31. Lown RN, Philippe J, Navarro W, et al. Unrelated adult stem cell donor medical suitability: recommendations from the World Marrow Donor Association Clinical Working Group Committee. Bone Marrow Transplant. 2014;49(7):880–6.

    Article  CAS  Google Scholar 

  32. Schmidt CWP. Pediatric oncologic pharmacy: a complete guide to practice. Cham: Springer; 2019.

    Book  Google Scholar 

  33. Sacchi N, Costeas P, Hartwell L, et al. Haematopoietic stem cell donor registries: World Marrow Donor Association recommendations for evaluation of donor health. Bone Marrow Transplant. 2008;42:9–14.

    Article  CAS  Google Scholar 

  34. Kimura F, Kanda J, Ishiyama K, et al. ABO blood type incompatibility lost the unfavorable impact on outcome in unrelated bone marrow transplantation. Bone Marrow Transplant. 2019;54(10):1676–85. https://www.nature.com/articles/s41409-019-0496-2 (Aug 28).

    Article  CAS  Google Scholar 

  35. Schrauder A, Reiter A, Gadner H, et al. Superiority of allogeneic hematopoietic stem-cell transplantation compared with chemotherapy alone in high-risk childhood T-cell acute lymphoblastic leukemia: results from ALL-BFM 90 and 95. J Clin Oncol. 2006;24(36):5742–9.

    Article  Google Scholar 

  36. Lucchini G, Labopin M, Beohou E, et al. Impact of conditioning regimen on outcomes for children with acute myeloid leukemia undergoing transplantation in first complete remission. An analysis on Behalf of the Pediatric Disease Working Party of the European Group for Blood and Marrow Transplantation. Biol Blood Marrow Transplant. 2017;23(3):467–74.

    Article  CAS  Google Scholar 

  37. European Medicines Agency. Assessment report: Busilvex. https://www.ema.europa.eu/en/documents/variation-report/busilvex-h-c-472-ii-019-epar-assessment-report-variation_en.pdf 2014 (Oct 29, 2019).

  38. UCSF Benioff Children’s Hospital. Bone marrow transplant glossary. https://www.ucsfbenioffchildrens.org/education/bone_marrow_transplant_glossary/ (Oct 29, 2019).

  39. Harris AC, Boelens JJ, Ahn KW, et al. Comparison of pediatric allogeneic transplant outcomes using myeloablative busulfan with cyclophosphamide or fludarabine. Blood Adv. 2018;2(11):1198–206.

    Article  CAS  Google Scholar 

  40. Paczulla A, Rothfelder K, Raffel S, et al. O011 human acute myeloid leukemia stem cells selectively escape NKG2D-mediated NK cell control. Bone Marrow Transplant. 2019;54:16–141.

    Article  Google Scholar 

  41. Chaudhury S, Sparapani R, Hu Z, et al. Outcomes of allogeneic hematopoietic cell transplantation in children and young adults with chronic myeloid leukemia: a CIBMTR cohort analysis. Biol Blood Marrow Transplant. 2016;22(6):1056–64.

    Article  Google Scholar 

Download references

Author information

Authors and Affiliations

Authors

Rights and permissions

Reprints and permissions

Copyright information

© 2020 Springer Nature Switzerland AG

About this chapter

Check for updates. Verify currency and authenticity via CrossMark

Cite this chapter

Schmidt, C.W.P. (2020). Stem Cell Transplantation in Pediatrics. In: Pediatric Hematopoietic Stem Cell Transplantation for Pharmacists. Springer, Cham. https://doi.org/10.1007/978-3-030-43491-5_3

Download citation

  • DOI: https://doi.org/10.1007/978-3-030-43491-5_3

  • Published:

  • Publisher Name: Springer, Cham

  • Print ISBN: 978-3-030-43490-8

  • Online ISBN: 978-3-030-43491-5

  • eBook Packages: MedicineMedicine (R0)

Publish with us

Policies and ethics