Successful Matched Related Bone Marrow Transplantation in a Patient with Autosomal Dominant Interferon Gamma Receptor 1
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LETTER TO EDITOR
Successful Matched Related Bone Marrow Transplantation in a Patient with Autosomal Dominant Interferon Gamma Receptor 1 Deficiency Christa S. Zerbe 1 & Dimana Dimitrova 2 & Juan J. Gea-Banacloche 3 & Samantha Kreuzburg 1 & Steven M. Holland 1 & Jennifer A. Kanakry 2 Received: 27 May 2020 / Accepted: 9 July 2020 # This is a U.S. Government work and not under copyright protection in the US; foreign copyright protection may apply 2020
Abstract Summary This is a report of a successful bone marrow transplant in an IFN-γR1 patient with progressive mycobacterial infection. Purpose Hematopoietic cell transplant in patients with interferon gamma receptor deficiencies has been fraught with challenges, not the least of which is failure of engraftment and infectious complications. Methods This is a report of a successful hematopoietic cell transplant in an actively infected patient of advanced age. Results This case report shows successful engraftment and resolution of infection posttransplant using a matched related donor in a single institution. Conclusion A successful curative HCT despite persistent, disseminated, nontuberculous mycobacterial infection in a patient with AD-IFNγR1 suggests that this approach, while difficult, may be useful in other patients with otherwise refractory disease. Keywords Interferon gamma receptor . hematopoietic cell transplant . nontuberculous mycobacteria . infection
To the Editor The interferon-γ pathway plays a critical role in the innate response to intracellular infection. The increased susceptibility to mycobacterial infections has been shown to be associated with defects in signaling through the interferon-γ receptor. Patients with autosomal dominant interferon gamma receptor 1 deficiency (AD-IFNγR1) have reduced signaling through the interferon-γ receptor leading to susceptibility to nontuberculous mycobacteria (NTM), among other organisms. In contrast to patients with complete IFNγ receptor deficiency who present in infancy and early childhood, patients with autosomal dominant IFNγR1 deficiency typically
* Christa S. Zerbe [email protected] 1
Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, MD, USA
2
Experimental Transplantation and Immunology Branch, National Cancer Institute, National Institutes of Health, Bethesda, MD, USA
3
Mayo Clinic, Phoenix, AZ, USA
present in later childhood through early adulthood with infection. Hematopoietic cell transplantation (HCT) is potentially curative yet historically very difficult in the complete receptor deficiency population [1–6]. Roesler et al. looked at eight patients with complete IFN-γR1 deficiency who received a total of 11 HCTs, mostly myeloablative, 10 from matched related donors, and 1 from an HLA-haploidentical parent. They found only 3 curative outcomes with the others characterized by primary or secondary graft failure or high mortality [1]. Much of the high mortality was related to mycobacterial infection
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