BMT for Adults with CGD

Advances over the last ten years have now made it possible for doctors to consider treating adults affected by CGD by bone marrow transplantation (BMT).

BMT is a specialised procedure and should be done by skilled and experienced transplant teams working in specialist centres.  The decision to proceed to BMT is based on a person’s clinical features, their infectious and non-infectious complications and anticipated clinical course without transplantation.  Overall survival rates for adult patients are currently greater than 76% (Chiesa et al; 2020) and result in a reduction in CGD-related complications.  However, the risks of transplant are increased considerably in patients who have already sustained significant damage to their vital organs (for example to the gut or lungs) and in CGD patients with uncontrolled infection or inflammation.  The type of BMT donor available and extent of tissue match also influences patient outcomes.

The BMT procedure itself carries risks of mortality and complications post BMT (for example graft versus host disease), and these must be weighed against the potential longer-term survival benefits and opportunity for cure, when considering whether a BMT is suitable for an individual patient. Patients therefore undergo careful selection by multidisciplinary transplant teams who will discuss a patient’s risk profile.

If you are considering a BMT as an adult CGD patient, you, and your family you will be given the opportunity to meet members of the transplant team, visit the transplant unit and discuss worries and ask questions about the procedure and the risks involved.

Useful resources:

Key questions to ask when considering a BMT.  This leaflet gives a list of questions that we recommend you ask before considering a BMT at a particular centre.

NHS commissioning policy

https://www.england.nhs.uk/commissioning/wp-content/uploads/sites/12/2019/07/Clinical-Commissioning-Policy_Allogeneic-Haematopoietic-Stem-Cell-Transplant-for-Primary-Immunodeficiencies.pdf

Related articles:

International study of BMT outcomes provides a wealth of data to assess individual risk for CGD transplants

UK Adult BMT Success for CGD Patients emphasises need for transplants to be done in highly specialised centres

Published data:

Haematopoietic Cell Transplantation in Chronic Granulomatous Disease: a Study on 712 Children and Adults. Robert Chiesa, Junfeng Wang, Henric-Jan Blok, Sheree Hazelaar, Benedicte Neven, Despina Moshous, Ansgar S Schulz, Manfred Hoenig, Fabian Hauck, Amal Al Seraihy, Jolanta Gozdzik, Per T Ljungman, Caroline Lindemans, Juliana Fernandes, Krzysztof Kalwak Brigitte Strahm, Urs Schanz, Petr Sedlacek, Karl-Walter Sykora, Serap Aksoylar, Franco Locatelli, Polina Stepensky, Robert F Wynn, Su Han Lum, Marco Zecca, Fulvio Porta, Mervi H Taskinen, Brenda Es Gibson, Susanne Matthes-Martin, Musa Karakukcu, Mathias M Hauri-Hohl, Paul Veys, Andrew R Gennery, Giovanna Lucchini, Matthias Felber, Michael H Albert, Dmitry Balashov, Arjan C Lankester, Tayfun Güngör, Mary Slatter.

Blood 2020 Jul 2; blood.2020005590. doi: 10.1182/blood.2020005590. Online ahead of print.

Successful outcome following allogeneic hematopoietic stem cell transplantation in adults with primary immunodeficiency.
Fox TA, Chakraverty R, Burns S, Carpenter B, Thomson K, Lowe D, Fielding A, Peggs K, Kottaridis P, Uttenthal B, Bigley V, Buckland M, Grandage V, Denovan S, Grace S, Dahlstrom J, Workman S, Symes A, Mackinnon S, Hough R, Morris E.
Blood. 2017 Dec 26. pii: blood-2017-09-807487. doi: 10.1182/blood-2017-09-807487. [Epub ahead of print]

Reduced-intensity conditioning and HLA-matched haemopoietic stem-cell transplantation in patients with chronic granulomatous disease: a prospective multicentre study.

Güngör T, Teira P, Slatter M, Stussi G, Stepensky P, Moshous D, Vermont C, Ahmad I, Shaw PJ, Telles da Cunha JM, Schlegel PG, Hough R, Fasth A, Kentouche K, Gruhn B, Fernandes JF, Lachance S, Bredius R, Resnick IB, Belohradsky BH, Gennery A, Fischer A, Gaspar HB, Schanz U, Seger R, Rentsch K, Veys P, Haddad E, Albert MH, Hassan M; Inborn Errors Working Party of the European Society for Blood and Marrow Transplantation. Lancet. 2014 Feb 1;383(9915):436-48. doi: 10.1016/S0140-6736(13)62069-3. Epub 2013 Oct 23. PMID: 24161820

Reviewed November 2020