This annual conference on bone marrow transplantation brings together patients, families, clinicians and nurses from across Europe.
This year the CGD Research Trust was represented by our CGD specialist nurse, Rhian Lant. 'It was a good opportunity to spread awareness of CGD and the work of the CGD Research Trust. The meeting was very well attended and the sessions covered topics such as managing infections in BMT patients, new bone marrow conditioning treatments, better treatments and prevention of graft versus host disease and the increasing use of cord blood as a source of stem cells for transplantation. It was a great learning experience for me', said Rhian.
At the meeting Rhian presented a poster examining the decision processes involved for an adult CGD patient in choosing to go forward for a bone marrow transplant. Rhian explains: 'Today's adult patients are facing a changed and changing future to the one they thought they had when diagnosed as a child. Looking again at the BMT procedure has been enlightening for this family. When they looked at BMT again, they found their knowledge was completely out of date.' Taking this step has meant that all members of the famiy have evaluated their own place in a family with a genetic diosrder, and as the relative of a person with CGD. This piece reflects the differences, struggles and importance of every family member.
At the meeting there were several other presentations and posters that covered CGD. One poster described a Swedish couple's decision to use in vitro fertilisation and preimplantation genetic diagnosis (PGD) to obtain a tissue match for their son who had X-linked CGD and who could not find a suitably matched donor. The couple had to go to Brussels for the treatment, as PGD is illegal in Sweden. Two PGD cycles were needed to select healthy embryos that were a perfect tissue match. The second cycle resulted in a healthy girl. Cord blood was collected at birth but there were insufficient cells to treat her CGD affected brother and they waited twelve months when a conventional bone marrow transplant was performed. The transplant was successful. The boy's infections were resolved and he is doing well.
The BMT team at Newcastle presented their centre's excellent results from treating 20 CGD patients between 1998 and 2007 with haematopoietic stem cell transplantation (HSCT). The procedure was used for people between the ages of 15 months and 21 years and used stem cells from sibling or matched unrelated donors. Fifteen of these patients received bone marrow, three received peripheral blood stem cells and two received stem cells from cord blood. The centre reported a 90% survival rate with functioning neutrophils, resolution of infections and impressive catch-up growth. Eighty four percent of patients treated had either no or mild graft versus host disease. The two patients with significant graft versus host disease had previous infection and inflammation and 13 of the 18 survivors are off prophylactic medication and have excellent quality of life. Dr Gennery, who headed the study comments 'This study shows that HSCT is curative for CGD and should certainly be considered early in the treatment of CGD if is there is a suitable donor available. We are learning so much more of the risks for treating CGD patients by HSCT and know that the complications of the treatment are less frequent and the outcome better in those patients that don't have a pre-existing infection or inflammatory problems. The implications are that as advances are made more people with CGD will be treated by HSCT and cured of their condition.'
IMPORTANT NOTE :
The information contained on this website is intended only as a guideline, not as a substitute for medical advice. Always consult your doctor if you or your child has any CGD symptoms or concerns.
© 2001-2007 The Chronic Granulomatous Disorder (CGD) Research Trust
Registered Charity No. 1003425 email:cgd@cgdrt.co.uk
The CGD Research Trust is a member of the Association of Medical Research Charities (AMRC), the Genetic Interest Group (GiG) and an associate member of the International Patient Organisation of Primary Immunodeficiencies (IPOPI)
Site Map | Terms and Conditions | Privacy
