Haematopoietic cell transplantation (HCT) has become standard of care for an increasing number of inborn errors of immunity (IEI). This is the first report to compare the transplant outcomes according to T-replete HLA-matched grafts using alemtuzumab (n=117) and T-depleted HLA-mismatched grafts using TCR αβ/CD19 depletion (n=47) in children with IEI who underwent first HCT between 2014 and 2019. All patients received treosulfan-based conditioning except patients with DNA repair disorders. For T-replete grafts, the stem cell source was marrow in 25 (21%) patients, PBSC in 85 (73%) and CB in 7 (6%). TCR αβ/CD19 depletion was performed on PBSC from 45 haploidentical parental donors and 2 mismatched unrelated donors. The 3-year OS and EFS for the entire cohort were 85% (77-90%) and 79% (69-86%) respectively. Analysis by age at transplant revealed a comparable 3-year OS between T-replete grafts (88%, 76-94%) and T-depleted grafts (87%, 64-96%) in younger patients (<5 years of age at HCT). For older patients more than 5 years of age, the OS was significantly lower in T-depleted grafts (55%, 23-78%), compared to T-replete grafts (87%, 68-95%) (p=0.03). Grade III-IV aGvHD was observed in 8% of T-replete marrow, 7% of T-replete PBSC, 14% of T-replete CB and 2% of T-depleted PBSC (p=0.73). Higher incidence of viraemia (p<0.001) and delayed CD3 reconstitution (p=0.003) were observed after T-depleted graft HCT. These data indicate that mismatched donor transplant after TCR αβ and CD19 depletion represents an excellent alternative for younger children with IEI in need of an allograft.
Disappearance Of L-Dopa SIR,-Since your leading article (24 June 1967, p. 783) on the successful use of dopa in the treatment of Parkinsonism, as previously reported in the New England fournal of Medicine,' there have been other reports of the efficiency of this mode of treatment. It has been glowingly reported by Alistair Cooke in the Guardian (3 February 1969) and splashed across the pages of Life magazine (11 October 1968). A controlled trial has recently been done in this country in postencephalitic Parkinsonism and confirms the usefulness of the drug.' Unfortunately this drug has always been very difficult to obtain in this country, and in the last few months it has virtually disappeared. This is a disgraceful situation that is hard for a doctor to understand, but it seems to be based on the apathy of some drug houses and curious takeover activities behind the scenes, somewhat reminiscent of the Third Man, by others. Is it not time that the British pharmaceutical industry was mobilized to serve a pressing need ?-I am, etc., K. W. G. HEATHFIELD.
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