Access through your institution Buy or subscribe Allogeneic hematopoietic SCT is currently the only procedure capable of curing children with MHC class II deficiency.1, 2, 3, 4 However, the

survival rate is much lower than in other primary immunodeficiency disorders (PID), with graft failure and GVHD being the major causes of treatment failure.2, 4 The optimal approach to

hematopoietic SCT in children with PID who lack a matched sibling donor is controversial.5 Some centers continue to use myeloablative conditioning regimen in children with PID who undergo

HLA-mismatched related T-cell-depleted BMT associated with high risk of graft failure.2, 5 However, recent success with reduced-intensity conditioning regimen before unrelated BMT shows that

cord blood units and the higher frequency of rare haplotypes compared with BM registries.8, 9, 10 In September 2004, a 1-month-old boy with the diagnosis of MHC class II deficiency was

referred to our BMT Service. Previously, his two brothers and one sister had died of recurrent infections in their hometown before the age of 7 months. A fourth child was born, and she was

diagnosed in our hospital with MHC class II deficiency. She underwent a T-cell-depleted haploidentical BMT in another institution, but died of a severe acute GVHD. This family belongs to

Kolla Amerindians, an ethnic minority from the far northwest of Argentina. During the neonatal period, the boy had received BCG immunization without any complications. After his evaluation,

a T-cell-depleted haploidentical BMT from his mother was performed. Conditioning consisted of BU (20 mg/kg), CY (200 mg/kg) and rabbit antithymocyte globulin (30 mg/kg). The graft was

T-cell-depleted by means of soybean agglutination and E-rosetting. CsA was administered for GVHD prophylaxis. On day +7, he developed acute skin and intestinal GVHD, which resolved with

methylprednisolone. On day +43, grade III acute intestinal and liver GVHD were confirmed by biopsies; tacrolimus was therefore initiated after CsA was discontinued. On day +56, the boy

developed congestive heart failure, and an echocardiography showed hypertrophic cardiomyopathy. Thus, despite the improvement in clinical manifestations and laboratory findings, tacrolimus

was stopped and methylprednisolone was continued at a dose of 2 mg/kg/day. On day +83, the GVHD resolved and the cardiomyopathy had regressed completely. DR expression on monocytes and B

lymphocytes was only transitorily detected during the first 6 months post transplantation (Table 1). This is a preview of subscription content, access via your institution ACCESS OPTIONS

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institutional subscriptions * Read our FAQs * Contact customer support REFERENCES * Casper JT, Ash RA, Kirchner P, Hunter JB, Havens PL, Chusid MJ . Successful treatment with an

unrelated-donor bone marrow transplant in an HLA-deficient patient with severe combined immune deficiency (‘bare lymphocyte syndrome’). _J Pediatr_ 1990; 116: 262–265. Article  CAS  PubMed 

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INFORMATION AUTHORS AND AFFILIATIONS * Bone Marrow Transplantation Service, Hospital de Pediatría ‘Prof Dr Juan P Garrahan’, Buenos Aires, Argentina M Bonduel, R Staciuk & C Figueroa *

Immunology Service, Hospital de Pediatría ‘Prof Dr Juan P Garrahan’, Buenos Aires, Argentina M Oleastro & J Rossi * Transfusion Medicine Service, Hospital de Pediatría ‘Prof Dr Juan P

Garrahan’, Buenos Aires, Argentina C Gamba & A del Pozo Authors * M Bonduel View author publications You can also search for this author inPubMed Google Scholar * R Staciuk View author

publications You can also search for this author inPubMed Google Scholar * C Figueroa View author publications You can also search for this author inPubMed Google Scholar * M Oleastro View

author publications You can also search for this author inPubMed Google Scholar * C Gamba View author publications You can also search for this author inPubMed Google Scholar * J Rossi View

author publications You can also search for this author inPubMed Google Scholar * A del Pozo View author publications You can also search for this author inPubMed Google Scholar

CORRESPONDING AUTHOR Correspondence to M Bonduel. RIGHTS AND PERMISSIONS Reprints and permissions ABOUT THIS ARTICLE CITE THIS ARTICLE Bonduel, M., Staciuk, R., Figueroa, C. _et al._

Unrelated cord blood transplantation and reduced-intensity conditioning regimen for graft failure in a child with Major Histocompatibility Complex class II deficiency. _Bone Marrow

Transplant_ 43, 817–818 (2009). https://doi.org/10.1038/bmt.2008.399 Download citation * Published: 15 December 2008 * Issue Date: May 2009 * DOI: https://doi.org/10.1038/bmt.2008.399 SHARE

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