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The Outcome of Allogeneic Hematopoietic Stem Cell Transplantation for Inherited Diseases Is Influenced by HLA Match, Year of Transplantation, and Immunized Female Donor

Ringden, Olle, MD, PhD1,2; Remberger, Mats, PhD3,4; Gustafsson, Britt, MD, PhD2,5; Moretti, Gianluca, BS1,2; Mattsson, Jonas, MD, PhD3,6; Winiarski, Jacek, MD, PhD2,5; Sadeghi, Behnam, MD, PhD1,2

doi: 10.1097/TP.0000000000002481
Original Clinical Science—General
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Background. For many inborn errors of metabolism (IEM), allogeneic hematopoietic stem cell transplantation (HSCT) is the only cure.

Methods. We report the outcome in 160 patients with inherited diseases, who were treated with HSCT in 3 decades. Median age was 3 years (range 0.1–63). Grafts were from matched related donors (MRDs, 56), matched unrelated donors (MUDs, 66), or HLA-mismatched donors (38).

Results. Graft failure (GF) occurred in 26 patients (16%), severe acute graft-versus-host disease (GVHD) in 9 (6%), and chronic GVHD in 23 (12%). Ten-year survival was 64% before the year 2000 and 86% after that (P = 0.01). Ten-year survival for MRD grafts was 90%, as opposed to 79% for MUD grafts and 56% for HLA-mismatched grafts (P = 0.03). In multivariate analysis, GF was associated with having an HLA-mismatched donor (P < 0.05) or MUD (P = 0.015) and with reduced-intensity conditioning (P < 0.01). Death was associated with year of transplant (P = 0.015), having an HLA-mismatched donor (P < 0.001), and being a male recipient from an immune female donor (P = 0.05).

Conclusions. The outcome after HSCT for IEM depends on HLA match, year and immune female donor.

1 Translational Cell Therapy Research, Karolinska Institutet, Karolinska University Hospital, Huddinge, Stockholm, Sweden.

2 Department of Clinical Science, Intervention and Technology, Karolinska Institutet, Karolinska University Hospital, Huddinge, Stockholm, Sweden.

3 Department of Oncology and Pathology, Karolinska Institutet, Karolinska University Hospital, Huddinge, Stockholm, Sweden.

4 Department of Medical Sciences, Uppsala University, and KFUE, Uppsala university hospital, Uppsala, Sweden.

5 Department of Pediatrics, Karolinska Institutet, Karolinska University Hospital, Huddinge, Stockholm, Sweden.

6 Department of Clinical Immunology, Karolinska Institutet, Karolinska University Hospital, Huddinge, Stockholm, Sweden.

Received 19 June 2018. Revision received 24 September 2018.

Accepted 2 October 2018.

The authors declare no funding or conflicts of interests.

O.R. initiated the study, contributed patients, designed the study and wrote the first draft. M.R. collected data, did statistical analysis and figures, designed the study and contributed in writing. B.G., J.W., and J.M. contributed patients. All authors contributed in analysis, interpretation, writing and approved the final article.

Correspondence: Olle Ringden, MD, PhD, Translational Cell Therapy Research Group (TCR), Karolinska Institutet, Kliniskt Forskningscentrum, NOVUM, Plan 6, Hälsovägen 7-9, 141 57 Huddinge, Sweden. (olle.ringden@ki.se).

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