The Journal of Pediatrics
Volume 154, Issue 5 , Pages 766-768 , May 2009

Homozygous α-Thalassemia Treated with Intrauterine Transfusions and Unrelated Donor Hematopoietic Cell Transplantation

  • Joanna S. Yi, MD

      Affiliations

    • Department of Pediatrics, University of Minnesota, Minneapolis, MN
    • ,
  • Christopher L. Moertel, MD

      Affiliations

    • Division of Pediatric Hematology/Oncology Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN
    • Corresponding Author InformationReprint requests: Christopher L. Moertel, MD, Mayo Mail Code 484/420 Delaware St SE, Minneapolis, MN 55455
    • ,
  • K. Scott Baker, MD

      Affiliations

    • Division of Pediatric Hematology/Oncology Blood and Marrow Transplantation, University of Minnesota, Minneapolis, MN

Received 12 June 2008 ,Revised 3 October 2008 ,Accepted 17 November 2008.

References 

  1. Carr S, Rubin L, Dixon D, Star J, Dailey J. Intrauterine therapy for homozygous α-thalassemia. Obstet Gynecol. 1995;85:876–879
  2. Westgren M, Ringden O, Eik-Nes S, Ek S, Anvret M, Brubakk AM, et al. Lack of evidence of permanent engraftment after in utero fetal stem cell transplantation in congenital hemoglobinopathies. Transplantation. 1996;61:1176–1179
  3. Fung TY, Lau TK, Kohng LC, Keung LC. Homozygous alpha-thalassemia associated with hypospadias in three survivors. Am J Med Genet. 1999;82:225–227
  4. Sohan K, Billington M, Pamphilon D, Goulden N, Kyle P. Normal growth and development following in utero diagnoses and treatment of homozygous α-thalassemia. BJOG. 2002;109:1308–1310
  5. Joshi D-D, Nickerson HJ, McManus MJ. Hydrops fetalis caused by homozygous α-thalassemia and Rh Antigen allloimmunization. Clin Med Res. 2004;2:228–232
  6. Thornley I, Lehmann L, Ferguson WS, Davis I, Forman EN, Guinan EC. Homozygous α-thalassemia treated with intrauterine transfusions and postnatal hematopoietic stem cell transplantation. Bone Marrow Transplant. 2003;32:341–342
  7. Cowan MJ, Golbus M. In utero hematopoietic stem cell transplants for inherited diseases. Am J Pediatr Hematol Oncol. 1994;16:35–42
  8. Eddleman K, Hobbins J, Hayward A, Migliaccio AR, Skupsi D, Zervoudakis I, et al. In utero paternal t-depleted marrow stem cells for the treatment of homozygous a-thalassemia: a case report. Stem Cell Therapy of Inherited Hemapoietic Disorders. 1998;27:57–58
  9. Hayward A, Ambruso D, Battaglia F, Donlon T, Eddelman K, Giller R, et al. Microchimerism and tolerance following intrauterine transplantation and transfusion for α-thalassemia-1. Fetal Diagn Ther. 1998;13:8–14
  10. Chik K, Shing MM, Li C, Leung TF, Tsang KS, Yuen HL, et al. Treatment of hemoglobin Bart's hydrops with bone marrow transplantation. J Pediatr. 1998;132:1039–1042
  11. Zhou X, Ha SY, Chan GC, Luk CW, Chan V, Haweekins B, et al. Successful mismatched sibling cord blood transplant in Hb Bart's disease. Bone Marrow Transplant. 2001;28:105–107
  12. La Nasa G, Argiolu F, Giardini C, Pession A, Fagioli F, Caocci G, et al. Unrelated bone marrow transplantation for beta-thalassemia patients: the experience of the Italian Bone Marrow Transplant Group. Ann NY Acad Sci. 2005;1054:186–195
  13. Lucarelli G, Galimberti M, Polchi P, Angelucci E, Baronciani D, Durazzi SM, et al. Bone marrow transplantation in thalassemia. Hematol Oncol Clin North Am. 1991;5:549–556

 The authors declare no conflicts of interest.

PII: S0022-3476(08)01040-8

doi: 10.1016/j.jpeds.2008.11.031

The Journal of Pediatrics
Volume 154, Issue 5 , Pages 766-768 , May 2009

Article Tools