A new retrospective study of severe combined immune deficiency (SCID) confirms that survival rates for patients are highest after a bone marrow transplant from a matched sibling donor – and identifies other biomarkers which predict overall survival.
A team of researchers analysed outcomes for 662 patients who had a haematopoietic cell transplantation as first-line treatment, one of the largest cohorts of SCID patients to date. Treatment took place between 1982 and 2012 in 33 hospitals in North America.
Their analysis showed that overall survival was highest following transplants from matched-sibling donors, compared to other donor types. For patients not receiving matched-sibling donation, survival was strongly linked to their SCID genotype, as well as their age and infection status at transplantation.
The researchers identified CD4+ cell counts at 6 and 12 months as prognostic biomarkers for overall survival and long-term immune reconstitution.
The research team, led by Dr Elie Haddad of the University of Montreal, Canada, write in the journal Blood that: "Our data emphasise the need for patient-tailored treatment strategies depending upon the underlying severe combined immune deficiency genotype.
"The prognostic significance of CD4+ cell counts as early as six months after haematopoietic cell transplant emphasises the importance of close follow-up of immune reconstitution to identify patients who may need additional intervention to prevent poor long-term outcome."
Dr Haddad adds: "We need to develop patient-specific treatment strategies. There is a critical need for neonatal screening to establish appropriate isolation, implement infection-prevention measures, particularly before transplantation, and ensure rapid referral for bone-marrow transplant or gene therapy after diagnosis."
The team call for further studies to identify patient- and transplant-related factors that affect early immune-system reconstitution.
Source: Haddad, E., Logan, B.R., Griffith, L.M., Buckley, R.H., Parrott, R.E., Prockop, S.E., Small, T.N., Chaisson, J., Dvorak, C.C., Murnane, M., Kapoor, N., Abdel-Azim, H., Hanson, I.C., Martinez, C., Bleesing, J.J.H., Chandra, S., Smith, A.R., Cavanaugh, M.E., Jyonouchi, S., Sullivan, K.E., Burroughs, L., Skoda-Smith, S., Haight, A.E., Tumlin, A.G., Quigg, T.C., Taylor, C., Dávila Saldaña, B.J., Keller, M.D., Seroogy, C.M., Desantes, K.B., Petrovic, A., Leiding, J.W., Shyr, D.C., Decaluwe, H., Teira, P., Gillio, A.P., Knutsen, A.P., Moore, T.B., Kletzel, M., Craddock, J.A., Aquino, V., Davis, J.H., Yu, L.C., Cuvelier, G.D.E., Bednarski, J.J., Goldman, F.D., Kang, E.M., Shereck, E., Porteus, M.H., Connelly, J.A., Fleisher, T.A., Malech, H.L., Shearer, W.T., Szabolcs, P., Thakar, M.S., Vander Lugt, M.T., Heimall, J., Yin, Z., Pulsipher, M.A., Pai, S.Y., Kohn, D.B., Puck, J.M., Cowan, M.J., O'Reilly, R.J., Notarangelo, L.D. (2018) “SCID genotype and 6-month post-transplant CD4 count predict survival and immune recovery”, Blood, available at doi: 10.1182/blood-2018-03-840702
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