Long-term survival and late deaths after hematopoietic cell transplantation for primary immunodeficiency diseases and inborn errors of metabolism.

Eapen, MaryAhn, Kwang WooOrchard, Paul JCowan, Morton JDavies, Stella MFasth, AndersHassebroek, AnnaAyas, MouhabBonfim, CarmemO'Brien, Tracey AGross, Thomas GHorwitz, MitchellHorwitz, EdwinKapoor, NeenaKurtzberg, JoanneMajhail, NavneetRingden, OlleSzabolcs, PaulVeys, PaulBaker, K Scott2022-03-232022-03-232012-091083-87911523-6536https://hdl.handle.net/10161/24685It is uncertain whether late mortality rates after hematopoietic cell transplantation for severe combined immunodeficiency (SCID), non-SCID primary immunodeficiency diseases (non-SCID PIDD), and inborn errors of metabolism (IEM) return to rates observed in the general population, matched for age, sex, and nationality. We studied patients with SCID (n = 201), non-SCID PIDD (n = 405), and IEM (n = 348) who survived for at least 2 years after transplantation with normal T cell function (SCID) or >95% donor chimerism (non-SCID PIDD and IEM). Importantly, mortality rate was significantly higher in these patients compared with the general population for several years after transplantation. The rate decreased toward the normal rate in patients with SCID and non-SCID PIDD beyond 6 years after transplantation, but not in patients with IEM. Active chronic graft-versus-host disease at 2 years was associated with increased risk of late mortality for all diseases (hazard ratio [HR], 1.87; P = .05). In addition, late mortality was higher in patients with non-SCID PIDD who received T cell-depleted grafts (HR 4.16; P = .007) and in patients with IEM who received unrelated donor grafts (HR, 2.72; P = .03) or mismatched related donor grafts (HR, 3.76; P = .01). The finding of higher mortality rates in these long-term survivors for many years after transplantation confirms the need for long-term surveillance.Transplantation ChimeraHumansMetabolism, Inborn ErrorsSevere Combined ImmunodeficiencyGraft vs Host DiseaseImmunologic Deficiency SyndromesChronic DiseaseTransplantation ConditioningHematopoietic Stem Cell TransplantationTransplantation, HomologousSurvival AnalysisTime FactorsInternational CooperationAdolescentChildChild, PreschoolInfantUnited StatesEuropeFemaleMaleUnrelated DonorsLong-term survival and late deaths after hematopoietic cell transplantation for primary immunodeficiency diseases and inborn errors of metabolism.Journal article2022-03-23