Skip to main content
SpringerLink
Log in

Non-Malignant Diseases Requiring Stem Cell Transplantation

  • Chapter
  • First Online:
Fertility Preservation in Oncological and Non-Oncological Diseases

  • 320 Accesses

Abstract

Haematopoietic stem cell transplantation (HSCT) is a potentially curative treatment option for several non-malignant diseases. Continuous progress in the field has led to broader indications for HSCT, increasing the number of patients (including children) being treated, and leading to a better overall long-term outcome. This chapter focuses on particularities of counselling in these patients, describes the gonadotoxicity of HSCT and disease-specific side effects such as iron overload in thalassemia major patients, toxicity of hydroxycarbamide in sickle cell disease, as well as comorbidities of these mostly hereditary and chronic diseases. In most HSCT in non-malignant diseases, there is enough time to perform fertility preservation procedures such as cryopreservation of gametes and gonadal tissue as well as GnRH agonists in females.

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

Access this chapter

Log in via an institution
Chapter
USD 29.95
Price excludes VAT (USA)
  • Available as PDF
  • Read on any device
  • Instant download
  • Own it forever
eBook
USD 84.99
Price excludes VAT (USA)
  • Available as EPUB and PDF
  • Read on any device
  • Instant download
  • Own it forever
Softcover Book
USD 109.99
Price excludes VAT (USA)
  • Compact, lightweight edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info
Hardcover Book
USD 109.99
Price excludes VAT (USA)
  • Durable hardcover 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. European Society for Blood and Marrow Transplantation. Annual report, 2017. Leiden: EBMT; 2017.

    Google Scholar 

  2. Passweg JR, Baldomero H, Peters C, Gaspar HB, Cesaro S, Dreger P, Duarte RF, Falkenburg JH, Farge-Bancel D, Gennery A, Halter J, Kröger N, Lanza F, Marsh J, Mohty M, Sureda A, Velardi A, Madrigal A. European Society for Blood and Marrow Transplantation EBMT. Hematopoietic SCT in Europe: data and trends in 2012 with special consideration of pediatric transplantation. Bone Marrow Transplant. 2014;49(6):744–50. https://doi.org/10.1038/bmt.2014.55.

    Article  PubMed  PubMed Central  Google Scholar 

  3. Dalle JH, Lucchini G, Balduzzi A, Ifversen M, Jahnukainen K, Macklon KT, Ahler A, Jarisch A, Ansari M, Beohou E, Bresters D, Corbacioglu S, Dalissier A, Diaz de Heredia Rubio C, Diesch T, Gibson B, Klingebiel T, Lankester A, Lawitschka A, Moffat R, Peters C, Poirot C, Saenger N, Sedlacek P, Trigoso E, Vettenranta K, Wachowiak J, Willasch A, von Wolff M, Yaniv I, Yesilipek A, Bader P. State-of-the-art fertility preservation in children and adolescents undergoing haematopoietic stem cell transplantation: a report on the expert meeting of the Paediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT) in Baden, Austria, 29-30 September 2015. Bone Marrow Transplant. 2017;52(7):1029–35. https://doi.org/10.1038/bmt.2017.21.

    Article  PubMed  Google Scholar 

  4. Balduzzi A, Dalle JH, Jahnukainen K, von Wolff M, Lucchini G, Ifversen M, Macklon KT, Poirot C, Diesch T, Jarisch A, Bresters D, Yaniv I, Gibson B, Willasch AM, Fadini R, Ferrari L, Lawitschka A, Ahler A, Sänger N, Corbacioglu S, Ansari M, Moffat R, Dalissier A, Beohou E, Sedlacek P, Lankester A, De Heredia Rubio CD, Vettenranta K, Wachowiak J, Yesilipek A, Trigoso E, Klingebiel T, Peters C, Bader P. Fertility preservation issues in pediatric hematopoietic stem cell transplantation: practical approaches from the consensus of the pediatric diseases working party of the EBMT and the international BFM study group. Bone Marrow Transplant. 2017;52(10):1406–15. https://doi.org/10.1038/bmt.2017.147.

    Article  PubMed  CAS  Google Scholar 

  5. Borgmann-Staudt A, Rendtorff R, Reinmuth S, Hohmann C, Keil T, Schuster FR, Holter W, Ehlert K, Keslova P, Lawitschka A, Jarisch A, Strauss G. Fertility after allogeneic haematopoietic stem cell transplantation in childhood and adolescence. 2011; https://doi.org/10.1038/bmt.2011.78.

  6. Zynda A, Reinmuth S, Pfitzer C, Hohmann C, Keil T, Borgmann-Staudt A. Childhood leukemia and its impact on graduation and having children: results from a national survey. Leuk Lymphoma. 2012;53(12):2419–22. https://doi.org/10.3109/10428194.2012.688965.

    Article  PubMed  Google Scholar 

  7. Matthews SJ, Picton H, Ernst E, Andersen CY. Successful pregnancy in a woman previously suffering from β-thalassemia following transplantation of ovarian tissue cryopreserved before puberty. Minerva Ginecol. 2018;70(4):432–5. https://doi.org/10.23736/S0026-4784.18.04240-5.

    Article  PubMed  Google Scholar 

  8. Dvorak CC, Gracia CR, Sanders JE, Cheng EY, Scott Baker K, Pulsipher MA, Petryk A. NCI, NHLBI/PBMTC first international conference on late effects after pediatric hematopoietic cell transplantation: endocrine challenges-thyroid dysfunction, growth impairment, bone health, & reproductive risks. Biol Blood Marrow Transplant. 2011;17:1725–38.

    Article  Google Scholar 

  9. Chow EJ, Stratton KL, Leisenring WM, Oeffinger KC, Sklar CA, Donaldson SS, Ginsberg JP, Kenney LB, Levine JM, Robison LL, Shnorhavorian M, Stovall M, Armstrong GT, Green DM. (2016). Pregnancy after chemotherapy in male and female survivors of childhood cancer treated between 1970 and 1999: a report from the Childhood Cancer Survivor Study cohort. Lancet Oncol. 2016 May;17(5):567-576. oi: https://doi.org/10.1016/S1470-2045(16)00086-3.

  10. Pfitzer C, Orawa H, Balcerek M, Langer T, Dirksen U, Keslova P, Zubarovskaya N, Schuster FR, Jarisch A, Strauss G, Borgmann-Staudt A. Dynamics of fertility impairment and recovery after allogeneic haematopoietic stem cell transplantation in childhood and adolescence: results from a longitudinal study. J Cancer Res Clin Oncol. 2015;141(1):135–42. https://doi.org/10.1007/s00432-014-1781-5.

    Article  PubMed  CAS  Google Scholar 

  11. Sanders JE, Hawley J, Levy W, Gooley T, Buckner CD, Deeg HJ, Doney K, Storb R, Sullivan K, Witherspoon R, Appelbaum FR. Pregnancies following high-dose cyclophosphamide with or without high-dose busulfan or total-body irradiation and bone marrow transplantation. Blood. 1996;87:3045–52.

    Article  CAS  Google Scholar 

  12. Vatanen A, Wilhelmsson M, Borgström B, Gustafsson B, Taskinen M, Saarinen-Pihkala UM, Winiarski J, Jahnukainen K. Ovarian function after allogeneic hematopoietic stem cell transplantation in childhood and adolescence. Eur J Endocrinol. 2014;170(2):211–8. https://doi.org/10.1530/EJE-13-0694.

    Article  PubMed  CAS  Google Scholar 

  13. Assouline E, Crocchiolo R, Prebet T, Broussais F, Coso D, Gamerre M, Vey N, Blaise D, Courbiere B. Impact of reduced-intensity conditioning allogeneic stem cell transplantation on women’s fertility. Clin Lymphoma Myeloma Leuk. 2013;13(6):704–10. https://doi.org/10.1016/j.clml.2013.05.014.

    Article  PubMed  Google Scholar 

  14. Panasiuk A, Nussey S, Veys P, Amrolia P, Rao K, Krawczuk-Rybak M, Leiper A. Gonadal function and fertility after stem cell transplantation in childhood: comparison of a reduced intensity conditioning regimen containing melphalan with a myeloablative regimen containing busulfan. Br J Haematol. 2015;170(5):719–26. https://doi.org/10.1111/bjh.13497.

    Article  PubMed  CAS  Google Scholar 

  15. Faraci M, Matthes-Martin S, Lanino E, Morreale G, Ferretti M, Giardino S, Micalizzi C, Balduzzi A. Two pregnancies shortly after transplantation with reduced intensity conditioning in chronic myeloid leukemia. Pediatr Transplant. 2016;20(1):158–61. https://doi.org/10.1111/petr.12630.

    Article  PubMed  CAS  Google Scholar 

  16. Yawn BP, Buchanan GR, Afenyi-Annan AN, Ballas SK, Hassell KL, James AH, Jordan L, Lanzkron SM, Lottenberg R, Savage WJ, Tanabe PJ, Ware RE, Murad MH, Goldsmith JC, Ortiz E, Fulwood R, Horton A, John-Sowah J. Management of sickle cell disease: summary of the 2014 evidence-based report by expert panel members. JAMA. 2014;312(10):1033–48. https://doi.org/10.1001/jama.2014.10517.

    Article  PubMed  CAS  Google Scholar 

  17. Wang WC, Ware RE, Miller ST, Iyer RV, Casella JF, Minniti CP, Rana S, Thornburg CD, Rogers ZR, Kalpatthi RV, Barredo JC, Brown RC, Sarnaik SA, Howard TH, Wynn LW, Kutlar A, Armstrong FD, Files BA, Goldsmith JC, Waclawiw MA, Huang X, Thompson BW. BABY HUG investigators. Hydroxycarbamide in very young children with sickle-cell anaemia: a multicentre, randomised, controlled trial (BABY HUG). Lancet. 2011;377(9778):1663–72. https://doi.org/10.1016/S0140-6736(11)60355-3.

    Article  PubMed  PubMed Central  CAS  Google Scholar 

  18. McGann PT, Ware RE. Hydroxyurea therapy for sickle cell anemia. Expert Opin Drug Saf. 2015;14(11):1749–58. https://doi.org/10.1517/14740338.2015.1088827.

    Article  PubMed  PubMed Central  CAS  Google Scholar 

  19. Elchuri SV, Williamson RS, Clark Brown R, Haight AE, Spencer JB, Buchanan I, Hassen-Schilling L, Brown MR, Mertens AC, Meacham LR. The effects of hydroxyurea and bone marrow transplant on anti-Müllerian hormone (AMH) levels in females with sickle cell anemia. Blood Cells Mol Dis. 2015;55(1):56–61. https://doi.org/10.1016/j.bcmd.2015.03.012.

    Article  PubMed  CAS  Google Scholar 

  20. Matthews M, Pollack R. (2017). Acute pain crisis in a patient with sickle cell disease undergoing ovarian simulation for fertility preservation prior to curative stem cell transplantation: case report and literature review. J Assist Reprod Genet. Nov;34(11):1445-1448.doi: https://doi.org/10.1007/s10815-017-1008-1.

  21. Dovey S, Krishnamurti L, Sanfilippo J, Gunawardena S, Mclendon P, Campbell M, Alway S, Efymow B, Gracia C. Oocyte cryopreservation in a patient with sickle cell disease prior to hematopoietic stem cell transplantation: first report. J Assist Reprod Genet. 2012;29(3):265–9. https://doi.org/10.1007/s10815-011-9698-2.

    Article  PubMed  PubMed Central  Google Scholar 

  22. Smith-Whitley K. Reproductive issues in sickle cell disease. Blood. 2014;124(24):3538–43. https://doi.org/10.1182/blood-2014-07-577619.

    Article  PubMed  CAS  Google Scholar 

  23. Schyrr F, Dolci M, Nydegger M, Canellini G, Andreu-Ullrich H, Joseph JM, Diezi M, Cachat F, Rizzi M, Renella R. Perioperative care of children with sickle cell disease: a systematic review and clinical recommendations. Am J Hematol. 2019;95(1):78–96. https://doi.org/10.1002/ajh.25626.

    Article  PubMed  Google Scholar 

  24. Fung EB, Harmatz PR, Lee PD, Milet M, Bellevue R, Jeng MR, Kalinyak KA, Hudes M, Bhatia S, Vichinsky EP, Multi-Centre Study of Iron Overload Research Group. Increased prevalence of iron-overload associated endocrinopathy in thalassaemia versus sickle-cell disease. Br J Haematol. 2006;135:574–82.

    Article  Google Scholar 

  25. Carlberg KT, Singer ST, Vichinsky EP. Fertility and pregnancy in women with transfusion-dependent thalassemia. Hematol Oncol Clin North Am. 2018;32(2):297–315. https://doi.org/10.1016/j.hoc.2017.11.004.

    Article  PubMed  Google Scholar 

  26. Skordis N, Gourni M, Kanaris C, Toumba M, Kleanthous M, Karatzia N, Pavlides N, Angastiniotis M. The impact of iron overload and genotype on gonadal function in women with thalassaemia major. Pediatr Endocrinol Rev. 2004;2(Suppl 2):292–5.

    PubMed  Google Scholar 

  27. Steward CG, Jarisch A. Haemopoietic stem cell transplantation for genetic disorders. Arch Dis Child. 2005;90:1259–63.

    Article  CAS  Google Scholar 

Download references

Author information

Authors and Affiliations

  1. Division of Stem Cell Transplantation and Immunology, Department for Children and Adolescents, University Hospital, Goethe University, Frankfurt am Main, Germany

    Andrea Jarisch

  2. Department of Gynaecological Endocrinology and Fertility Disorders, University Women’s Hospital Heidelberg, Heidelberg, Germany

    Ariane Germeyer

Authors
  1. Andrea Jarisch
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. Ariane Germeyer
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to Andrea Jarisch .

Editor information

Editors and Affiliations

  1. Division of Gynaecological Endocrinology and Reproductive Medicine, University Women’s Hospital of Bern, Bern, Switzerland

    Michael von Wolff

  2. Specialist Centre for Reproductive Medicine, Prenatal Medicine, Endocrinology and Osteology, amedes MVZ Hamburg, Hamburg, Germany

    Frank Nawroth

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

Jarisch, A., Germeyer, A. (2020). Non-Malignant Diseases Requiring Stem Cell Transplantation. In: von Wolff, M., Nawroth, F. (eds) Fertility Preservation in Oncological and Non-Oncological Diseases. Springer, Cham. https://doi.org/10.1007/978-3-030-47568-0_14

Download citation

  • DOI: https://doi.org/10.1007/978-3-030-47568-0_14

  • Published:

  • Publisher Name: Springer, Cham

  • Print ISBN: 978-3-030-47567-3

  • Online ISBN: 978-3-030-47568-0

  • eBook Packages: MedicineMedicine (R0)

Publish with us

Policies and ethics

Search

Navigation