In utero hematopoietic cell transplantation for hemoglobinopathies

Frontiers in Pharmacology

Volumn 5, Issue , 2015, Pages

  Derderian, S Christopher ^a   Jeanty, Cerine ^a   Walters, Mark C ^b   Vichinsky, Elliott ^b   MacKenzie, Tippi C ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b CHILDREN S HOSPITAL OAKLAND   (United States)

Author keywords

alpha thalassemia; chimerism; fetal therapy; in utero transplantation; tolerance

Indexed keywords

EID: 84975182269     PISSN: None     EISSN: 16639812     Source Type: Journal    
DOI: 10.3389/fphar.2014.00278     Document Type: Review

References (50)

1. Blakemore K. Hattenburg C. Stetten G. Berg K. South S. Murphy K. et al. (2004). In utero hematopoietic stem cell transplantation with haploidentical donor adult bone marrow in a canine model. Am. J. Obstet. Gynecol. 190 960–973. 10.1016/j.ajog.2004.01.014
2. Carr S. Rubin L. Dixon D. Star J. Dailey J. (1995). Intrauterine therapy for homozygous alpha-thalassemia. Obstet. Gynecol. 85 876–879. 10.1016/0029-7844(94)00334-A
3. Carrier E. Lee T. H. Busch M. P. Cowan M. J. (1995). Induction of tolerance in nondefective mice after in utero transplantation of major histocompatibility complex-mismatched fetal hematopoietic stem cells. Blood 86 4681–4690.
4. Chui D. H. Waye J. S. (1998). Hydrops fetalis caused by alpha-thalassemia: an emerging health care problem. Blood 91 2213–2222.
5. Cowan M. J. Golbus M. (1994). In utero hematopoietic stem cell transplants for inherited diseases. Am. J. Pediatr. Hematol. Oncol. 16 35–42.
6. Derderian S. C. Togarrati P. P. King C. Moradi P. W. Reynaud D. Czechowicz A. et al. (2014). In utero depletion of fetal hematopoietic stem cells improves engraftment after neonatal transplantation in mice. Blood 124 973–980. 10.1182/blood-2014-02-550327
7. Dwinnell S. J. Coad S. Butler B. Albersheim S. Wadsworth L. D. Wu J. K. et al. (2011). In utero diagnosis and management of a fetus with homozygous alpha-Thalassemia in the second trimester: a case report and literature review. J. Pediatr. Hematol. Oncol. 33 e358–e360. 10.1097/MPH.0b013e31821b368c
8. Flake A. W. Harrison M. R. Adzick N. S. Zanjani E. D. (1986). Transplantation of fetal hematopoietic stem cells in utero: the creation of hematopoietic chimeras. Science 233 776–778. 10.1126/science.2874611
9. Flake A. W. Roncarolo M. G. Puck J. M. Almeida-Porada G. Evans M. I. Johnson M. P. et al. (1996). Treatment of X-linked severe combined immunodeficiency by in utero transplantation of paternal bone marrow. N. Engl. J. Med. 335 1806–1810. 10.1056/NEJM199612123352404
10. Fleischman R. A. Mintz B. (1979). Prevention of genetic anemias in mice by microinjection of normal hematopoietic stem cells into the fetal placenta. Proc. Natl. Acad. Sci. U.S.A. 76 5736–5740. 10.1073/pnas.76.11.5736
11. Fucharoen S. Winichagoon P. Thonglairoam V. Siriboon W. Siritanaratkul N. Kanokpongsakdi S. et al. (1991). Prenatal diagnosis of thalassemia and hemoglobinopathies in Thailand: experience from 100 pregnancies. Southeast Asian J. Trop. Med. Public Health 22 16–29.
12. Ge H. Huang X. Li X. Chen S. Zheng J. Jiang H. et al. (2013). Noninvasive prenatal detection for pathogenic CNVs: the application in alpha-thalassemia. PLoS ONE 8:e67464. 10.1371/journal.pone.0067464
13. Golombeck K. Ball R. H. Lee H. Farrell J. A. Farmer D. L. Jacobs V. R. et al. (2006). Maternal morbidity after maternal-fetal surgery. Am. J. Obstet. Gynecol. 194 834–839. 10.1016/j.ajog.2005.10.807
14. Harrison M. R. Slotnick R. N. Crombleholme T. M. Golbus M. S. Tarantal A. F. Zanjani E. D. (1989). In-utero transplantation of fetal liver haemopoietic stem cells in monkeys. Lancet 2 1425–1427. 10.1016/S0140-6736(89)92036-9
15. Hayward A. Ambruso D. Battaglia F. Donlon T. Eddelman K. Giller R. et al. (1998). Microchimerism and tolerance following intrauterine transplantation and transfusion for alpha-thalassemia-1. Fetal Diagn. Ther. 13 8–14. 10.1159/000020793
16. Hoppe C. C. (2009). Newborn screening for non-sickling hemoglobinopathies. Hematology Am. Soc. Hematol. Educ. Program 19–25. 10.1182/asheducation-2009.1.19
17. Lam Y. H. Tang M. H. Lee C. P. Tse H. Y. (1999). Prenatal ultrasonographic prediction of homozygous type 1 alpha-thalassemia at 12 to 13 weeks of gestation. Am. J. Obstet. Gynecol. 180 148–150. 10.1016/S0002-9378(99)70165-1
18. Laros R. K. (1994). “Maternal hematologic disorders,” in Maternal-Fetal Medicine, Principles and Practice eds Creasy R. K. Resnick R. (Philadelphia, PA: WB Saunders), 905–933.
19. Lau Y. L. Chan L. C. Chan Y. Y. Ha S. Y. Yeung C. Y. Waye J. S. et al. (1997). Prevalence and genotypes of alpha- and beta-thalassemia carriers in Hong Kong – implications for population screening. N. Engl. J. Med. 336 1298–1301. 10.1056/NEJM199705013361805
20. Lee S. Y. Chow C. B. Li C. K. Chiu M. C. (2007). Outcome of intensive care of homozygous alpha-thalassaemia without prior intra-uterine therapy. J. Paediatr. Child Health 43 546–550. 10.1111/j.1440-1754.2007.01131.x
21. Leung W. C. Leung K. Y. Lau E. T. Tang M. H. Chan V. (2008). Alpha-thalassaemia. Semin. Fetal Neonatal Med. 13 215–22. 10.1016/j.siny.2008.02.006
22. Li Q. Wei J. Li D. (2007). Prenatal ultrasonographic prediction of homozygous alpha-thalassemia disease at midpregnancy. Int. J. Gynaecol. Obstet. 97 156–157. 10.1016/j.ijgo.2007.01.017
23. Lo Y. M. Tein M. S. Lau T. K. Haines C. J. Leung T. N. Poon P. M. et al. (1998). Quantitative analysis of fetal DNA in maternal plasma and serum: implications for noninvasive prenatal diagnosis. Am. J. Hum. Genet. 62 768–775. 10.1086/301800
24. Lucke T. Pfister S. Durken M. (2005). Neurodevelopmental outcome and haematological course of a long-time survivor with homozygous alpha-thalassaemia: case report and review of the literature. Acta Paediatr. 94 1330–1333. 10.1111/j.1651-2227.2005.tb02096.x
25. Merianos D. J. Tiblad E. Santore M. T. Todorow C. A. Laje P. Endo M. et al. (2009). Maternal alloantibodies induce a postnatal immune response that limits engraftment following in utero hematopoietic cell transplantation in mice. J. Clin. Invest. 119 2590–2600.
26. Migliaccio A. R. Papayannopoulou T. (2001). “Erythropoiesis,” in Disorders of Hemoglobin: Genetics, Pathophysiology, Clinical Management eds Steinberg M. H. Forget B. G. Higgs D. Nagel R. L. (Cambridge: Cambridge University Press), 52–71.
27. Misra M. V. Gutweiler J. R. Suh M. Y. Twark C. M. Valim C. Perez-Atayde A. et al. (2009). A murine model of graft-vs-host disease after in utero hematopoietic cell transplantation. J. Pediatr. Surg. 44 1102–1107; discussion 1107. 10.1016/j.jpedsurg.2009.02.033
28. Nicolaides K. Brizot Mde L. Patel F. Snijders R. (1994). Comparison of chorionic villus sampling and amniocentesis for fetal karyotyping at 10–13 weeks’ gestation. Lancet 344 435–439. 10.1016/S0140-6736(94)91769-8
29. Nijagal A. Derderian C. Le T. Jarvis E. Nguyen L. Tang Q. et al. (2013). Direct and indirect antigen presentation lead to deletion of donor-specific T cells after in utero hematopoietic cell transplantation in mice. Blood 121 4595–4602. 10.1182/blood-2012-10-463174
30. Nijagal A. Flake A. W. MacKenzie T. C. (2012). In utero hematopoietic cell transplantation for the treatment of congenital anomalies. Clin. Perinatol. 39 301–310. 10.1016/j.clp.2012.04.004
31. Nijagal A. Wegorzewska M. Jarvis E. Le T. Tang Q. MacKenzie T. C. (2011). Maternal T cells limit engraftment after in utero hematopoietic cell transplantation in mice. J. Clin. Invest. 121 582–592. 10.1172/JCI44907
32. No authors. (1976). Midtrimester amniocentesis for prenatal diagnosis. Safety and accuracy. JAMA 236 1471 – 1476. 10.1001/jama.1976.03270140023016
33. Owen R. D. (1945). Immunogenetic consequences of vascular anastomoses between bovine twins. Science 102 400–401. 10.1126/science.102.2651.400
34. Pearce R. D. Kiehm D. Armstrong D. T. Little P. B. Callahan J. W. Klunder L. R. et al. (1989). Induction of hemopoietic chimerism in the caprine fetus by intraperitoneal injection of fetal liver cells. Experientia 45 307–308. 10.1007/BF01951819
35. Peranteau W. H. Heaton T. E. Gu Y. C. Volk S. W. Bauer T. R. Alcorn K. et al. (2009). Haploidentical in utero hematopoietic cell transplantation improves phenotype and can induce tolerance for postnatal same-donor transplants in the canine leukocyte adhesion deficiency model. Biol. Blood Marrow Transplant. 15 293–305. 10.1016/j.bbmt.2008.11.034
36. Picus J. Aldrich W. R. Letvin N. L. (1985). A naturally occurring bone-marrow-chimeric primate. I. Integrity of its immune system. Transplantation 39 297–303. 10.1097/00007890-198503000-00018
37. Singer S. T. Styles L. Bojanowski J. Quirolo K. Foote D. Vichinsky E. P. (2000). Changing outcome of homozygous alpha-thalassemia: cautious optimism. J. Pediatr. Hematol. Oncol. 22 539–542. 10.1097/00043426-200011000-00014
38. Sirichotiyakul S. Charoenkwan P. Sanguansermsri T. (2012). Prenatal diagnosis of homozygous alpha-thalassemia-1 by cell-free fetal DNA in maternal plasma. Prenat. Diagn. 32 45–49. 10.1002/pd.2892
39. Sundberg K. Bang J. Smidt-Jensen S. Brocks V. Lundsteen C. Parner J. et al. (1997). Randomised study of risk of fetal loss related to early amniocentesis versus chorionic villus sampling. Lancet 350 697–703. 10.1016/S0140-6736(97)02449-5
40. Tarantal A. F. Goldstein O. Barley F. Cowan M. J. (2000). Transplantation of human peripheral blood stem cells into fetal rhesus monkeys (Macaca mulatta). Transplantation 69 1818–1823. 10.1097/00007890-200005150-00015
41. Touraine J. L. Raudrant D. Royo C. Rebaud A. Roncarolo M. G. Souillet G. et al. (1989). In-utero transplantation of stem cells in bare lymphocyte syndrome. Lancet 1:1382. 10.1016/S0140-6736(89)92819-5
42. van Dijk B. A. Boomsma D. I. de Man A. J. (1996). Blood group chimerism in human multiple births is not rare. Am. J. Med. Genet. 61 264–268. 10.1002/(SICI)1096-8628(19960122)61:3<264::AID-AJMG11>3.0.CO;2-R
43. Vichinsky E. P. (2009). Alpha thalassemia major–new mutations, intrauterine management, and outcomes. Hematology Am. Soc. Hematol. Educ. Program 35–41. 10.1182/asheducation-2009.1.35
44. Vrecenak J. D. Flake A. W. (2013). In utero hematopoietic cell transplantation–recent progress and the potential for clinical application. Cytotherapy 15 525–535. 10.1016/j.jcyt.2013.01.003
45. Vrecenak J. D. Pearson E. G. Santore M. T. Todorow C. A. Li H. Radu A. et al. (2014). Stable long-term mixed chimerism achieved in a canine model of allogeneic in utero hematopoietic cell transplantation. Blood 124 1987–1995. 10.1182/blood-2013-11-537571
46. Weisz B. Rosenbaum O. Chayen B. Peltz R. Feldman B. Lipitz S. (2009). Outcome of severely anaemic fetuses treated by intrauterine transfusions. Arch. Dis. Child Fetal Neonatal Ed. 94 F201–F204. 10.1136/adc.2008.143560
47. Wengler G. S. Lanfranchi A. Frusca T. Verardi R. Neva A. Brugnoni D. et al. (1996). In-utero transplantation of parental CD34 haematopoietic progenitor cells in a patient with X-linked severe combined immunodeficiency (SCIDXI). Lancet 348 1484–1487. 10.1016/S0140-6736(96)09392-0
48. Westgren M. Ringden O. Eik-Nes S. Ek S. Anvret M. Brubakk A. M. et al. (1996). Lack of evidence of permanent engraftment after in utero fetal stem cell transplantation in congenital hemoglobinopathies. Transplantation 61 1176–1179. 10.1097/00007890-199604270-00010
49. Yi J. S. Moertel C. L. Baker K. S. (2009). Homozygous alpha-thalassemia treated with intrauterine transfusions and unrelated donor hematopoietic cell transplantation. J. Pediatr. 154 766–768. 10.1016/j.jpeds.2008.11.031
50. Zhou X. Ha S. Y. Chan G. C. Luk C. W. Chan V. Hawkins B. et al. (2001). Successful mismatched sibling cord blood transplant in Hb Bart’s disease. Bone Marrow Transplant. 28 105–107. 10.1038/sj.bmt.1703104