![]() ![]() Total number of definitive cellular therapies performed over patient lifetime Need for subsequent therapy following first definitive cellular therapy HLA-mismatched other relative (haploidentical) ![]() Weight percentile at time of first cellular therapy Height percentile at time of first cellular therapy ![]() Infection at time of first cellular therapy Refer to the presence or absence of these features between date of ADA diagnosis and either start of ERT or first definitiveĬellular therapy (not necessarily whether they were still present at the onset of these treatments).ĮRT followed by gene therapy (ERT-GT) (N = 33)ĭuration of ERT before first definitive cellular therapy occurred (d)ĭuration of ERT before first definitive cellular therapy occurred (mo) ||Need for supplemental oxygen, CPAP, mechanical ventilation, weight and height percentiles, autoimmunity, and cardiac dysfunction Ada deficiency scid skin#Omenn syndrome: Generalized skin rash, no maternal lymphocytes, ≥80% of CD3 + or CD4 + T cells are CD45RO +, with at least 4 of 9 criteria being met including hepatomegaly, splenomegaly, lymphadenopathy, elevated IgE, elevated absoluteĮosinophil count, oligoclonal T cells, reduced PHA proliferation < 50%, hypomorphic mutation in known SCID gene, and low TRECs and/or CD4 +CD31 +CD45RA + and/or CD4 +CD45RA +CD62L + T cells. §Definition of SCID subtypes used in PIDTC 6901/6902 protocols based on published "Shearer criteria." 57 Typical SCID: CD3 + T cells 2 years old and ≤4 years old: 4 years old: <600 cells per cubic millimeter) and proliferation to PHA < 30% the lower limit of normal and no maternal T-cell engraftment. P value reflects comparison between patients receiving ERT only, GT, and HCT with or without pre-HCT ERT.ĬPAP, continuous positive airway pressure EF, ejection fraction NA, not applicable SF, shortening fraction. Baseline characteristics of patients with ADA-deficient SCID at the time of initial diagnosis ERT followed by gene therapy (ERT-GT) (N = 35) ![]()
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