Haploidentical stem cell transplantation with post-transplant cyclophosphamide in leukocyte adhesion deficiency type 1: a case report
- 41 Downloads
Leukocyte adhesion deficiency type 1 (LAD-1) is a rare autosomal recessive primary immunodeficiency disease (PID) that results from defective expression of the beta-2 integrin, CD18, on immune cells and results in impaired leukocyte adhesion, egression, and migration . Hematopoietic stem cell transplant (HSCT) is the only known curative approach for LAD-1 treatment . In the absence of a matched related donor (MRD) and matched unrelated donor (MUD), haploidentical HSCT using T cell replete graft with post-transplant cyclophosphamide (PT-CY) is an emerging option for patients with both malignant and benign hematological conditions [3, 4]. We report here the successful haploidentical transplantation using T cell replete graft and PT-CY in a patient with LAD-1.
Compliance with ethical standards
All procedures performed in studies involving human participants were in accordance with the ethical standards of the institutional and/or national research committee and with the 1964 Helsinki declaration and its later amendments or comparable ethical standards.
Conflict of interest
Was obtained from the patient’s parents in this study.
- 1.Movahedi M, Entezari N, Pourpak Z, Mamishi S, Chavoshzadeh Z, Gharagozlou M, Mir-Saeeid-Ghazi B, Fazlollahi MR, Zandieh F, Bemanian MH, Farhoudi A (2007) Clinical and laboratory findings in Iranian patients with leukocyte adhesion deficiency (study of 15 cases). J Clin Immunol 27:302–307CrossRefGoogle Scholar
- 2.Qasim W, Cavazzana-Calvo M, Davies EG, Davis J, Duval M, Eames G, Farinha N, Filopovich A, Fischer A, Friedrich W, Gennery A, Heilmann C, Landais P, Horwitz M, Porta F, Sedlacek P, Seger R, Slatten M, Teague L, Eapen M, Veys P (2009) Allogeneic hematopoietic stem-cell transplantation for leukocyte adhesion deficiency. Pediatrics 123:836–840CrossRefGoogle Scholar
- 4.Raiola AM, Dominietto A, di Grazia C, Lamparelli T, Gualandi F, Ibatici A, et al (2014) Unmanipulated haploidentical transplants compared with other alternative donors and matched sibling grafts. Biol Blood Marrow Transplant 20(10):1573–1579Google Scholar
- 7.Neven B, Diana JS, Castelle M, Magnani A, et al (2019) Haploidentical hematopoietic stem cell transplantation with post transplant cyclophosphamide for primary immunodeficiencies and inherited disorders in children. Biol Blood Marrow Transplant 25(7):1363–1373Google Scholar
- 8.Chiesa R, Gilmour K, Qasim W, Adams S, Worth AJJ, Zhan H, Montiel-Equihua CA, Derniame S, Cale C, Rao K, Hiwarkar P, Hough R, Saudemont A, Fahrenkrog CS, Goulden N, Amrolia PJ, Veys P (2012) Omission of in vivo T-cell depletion promotes rapid expansion of naive CD41 cord blood lymphocytes and restores adaptive immunity within 2 months after unrelated cord blood transplant. Br J Haematol 156(5):656–666CrossRefGoogle Scholar