FROM PULPCULTURE, 2 FEBRUARY 2004
I’d like to tell you all about a little boy I know who was born three days before my baby (back last August).
His name is Alexander. My wife nicknamed him Lockey (after his surname and it has stuck amongst his friends and family) He is very cute and is aged 6 months now. Over Christmas he developed a cold and couldn’t seem to get rid of it. After 3 weeks in hospital he got sent to Great Ormond Street Hospital. Great Ormond Street Hospital diagnosed that Lockey has no immune system (Severe Combined Immuno deficiency). This means the cold he caught, he cannot fight and get rid of. To this day he still has it. He is on oxygen feeds and a drip to try to increase his body weight. My daughter is about 25 lbs. Lockey is still only 12 lbs. Lockeys parents have some decisions ahead of them. I ask you to read two emails from his parents Carol and Colin…………
Well, we now know there are 5 potential donors (1 in the UK and 4 abroad) who are being contacted to come in for more tests. The consultant seemed disappointed by this as he hoped for around 10- 20 "broad matches".
Alexander needs some luck now as these 5 donors are only pre-matched against 6 of the 10 tissue markers required. They still need to match up against the other 4 tissue markers. With this in mind Carol and I are due for more tests this week to work out who will be more suitable as a donor should they use one of us. I can tell them this already but they insist on testing! My worry for Alexander is the severity of the chemotherapy required if Carol or I eventually donate bone marrow to him. Also, the initial recovery time in hospital goes up from approximately 6 weeks (for an unrelated donor match) to possibly 9 months. I don't want to think about this at the moment...
They have nearly tracked down the genetic defect in Carol which they would hope to correct with Gene Therapy when treating Alexander. So, gene therapy is starting to present itself as an option for Alexander - but this is yet to be confirmed. There are risks and benefits associated with both treatments (transplant or gene therapy) and we need to make a decision based on short, mid and long term risks. We will not be given more information until they know the unrelated donor situation. I understand this, as if we can find a suitable donor that is the best bet for Alexander.
Gene Therapy will mean Alex going in to a research situation - it isn't completely proven treatment yet as it is too new. However, I have found out that if it were unsuccessful he could have a transplant afterwards. But, I think there could be some long term risks we need to weigh up – likelihood of cancer, etc.
I have found out that bone marrow donation could mean (1) taking marrow from the hip under general anaesthetic (apparently painful afterwards) or (2) harvesting it from blood having been injected with hormones to help produce more stem cells. The later requires a set up a bit like a blood transfusion - they have got to be kidding! Either way I'll be unconscious, whether they knock me out or not...
Timewise I feel that he will be starting transplant treatment by
mid/late-March latest (as they are worried about his continued risk from infection) and if the unrelated donors don't match up he'll be starting chemotherapy by the end of February to take marrow from one of us. If this happens we will not get back home in March as we were hoping.
We are still staying in London in one of the Sick Children's Trust homes.
Latest email from them………
Alexander continues to improve everyday. He is still on oxygen and is
having continuous feeds to try and improve his weight. He is sitting up now and to be honest we sometimes forget he is so ill as he looks so well, except for the tubes up his nose.
Well this has been a good week in some ways as Alexander is now back up to the weight he was three months ago (6 Kilos or approx. 12 lbs.) before infection set in. So, some progress there.
We had a long conversation earlier in the week with our lead consultant and a professor of immunology regarding gene therapy. Alex is definitely a contender for this, but we need to get approval from an ethical body which controls such research. We asked them to go for approval whether or not we actually do decide to use this.
They are unhappy about giving Alexander any treatment which involves high levels of chemotherapy just in case the virus he still has on-board takes hold, this rules out some of the options available and a bone marrow transplant of whichever kind will probably only provide a partial immune system for him. Still, he could have additional treatment later on when he is older and stronger.
We think it is pretty unlikely that we will find a 10-out-of-10 unrelated bone marrow donor now, so the options are likely to be gene therapy or taking marrow from Carol or myself. We should know more about this in the very near future. But, either way, the intention is to start some form of treatment soon. Our target date is still 17th March, it could be sooner depending on the results of the donor searches still underway.
Full bone marrow transplant with a 10-out-of-10 unrelated donor needs
chemo. Risk of infertility. Should get a full recovery Half BMT from Carol or Colin with some chemo. Risk of infertility. Should
get a full recovery Half BMT from Carol or Colin with no Chemo. Will not get a full recovery.
Gene Therapy - No Chemo so no infertility - Should get a full recovery
Conundrum - A 10-out-of-10 unrelated donor usually means we will not be allowed to do the experimental gene therapy.
That's it for now. It is quite likely we will let you know exactly which treatment we will be using when we next write.
Have a good weekend.
Thanks for all the cards and text messages they are all much appreciated.
Lots of Love Carol and Colin
In the mean time if you are interested in becoming a bone marrow donor.
- Ideally you need to be male aged 18 to 30
- They will take women and older men if they are a good match (Don't let the local doctor put you off)
- The Bone marrow can be harvested with an operation OR via something similar to a Dialysis machine.
- If you are still interested contact the Anthony Nolan web site and
download a form to take to your local doctor.http://www.anthonynolan.org.uk