Xavier’s parents had taken along the negatives from St Luc’s and although the technique was different, according to Dr Kurtzberg the MRI was stable, the lumbar puncture good and no vital points had been affected. She found Xavier’s general health was good and she confirmed that the transplant should be done as quickly as possible because the chances of success IN XAVIER’S CASE, were 70% for an umbilical cord stem cell transplant.   The illness is widely known about in the United States, patients travelling there from all over the world. Between 7 and 14 children suffering from Krabbe’s disease are treated every year. To this day, 14 cases of the late-onset infant form have been transplanted between Duke (Dr Kurtzberg) and Minneapolis.   Furthermore, they visited the children’s hospital’s sterile unit where each child has his or her own room which has facilities enabling one parent to stay with the child 24 hours a day. That is part of the therapy and increases the likelihood of success. Generally one nurse is responsible for two patients, but if a child is very ill the nurse will look after that child only. Everything has been studied in the unit so that daily life resembles home life as far as possible: in the child’s room there is a fridge, a cooker, a bathroom, etc. There are also areas for the parents in the unit, such as a living room, a fully-equipped kitchen, a laundry room, etc.   Given their knowledge of the illness, their openness and receptiveness, their policy of keeping the parents informed and involved in the programme, there was only one choice to make for Xavier, which is that he should stay at Duke.   Unfortunately it isn’t as simple in practice. Xavier and his mum would have to be away from home for at least a year, whilst his little brother stayed in Belgium with his dad and grandma. Alexander would have to do without his mum for the whole time she was away because we wouldn’t be able to make frequent trips over there. Family life would be shattered, Alexander would take his first steps whilst far from his mum and during this time Xavier wouldn’t see his brother, his dad or his grandma… His mum would have to take a career break, and the rest of the family would have to make huge sacrifices in order to help as much as possible with the costs such a decision would imply (finding somewhere to live over there, living costs, medical expenses, etc.). It would be heartbreaking but if his parents had no other option, they would make any sacrifice if it meant saving Xavier.   Xavier’s parents were entirely convinced at this point that it was urgent to go ahead as soon as possible with a stem cell transplant but his mum wasn’t prepared to leave her child even for a single day, even in the sterile room, and it was impossible to make her change her mind about this.   Given the urgency if the situation, Xavier’s grandma contacted professor Vermylen at the Saint-Luc University Clinics, whose reputation in the field of transplants remains unmatched: “… Firstly the world premiere in 1986 of a marrow transplant in a child suffering from sickle cell anaemia. Since then about forty children have had transplants at St Luc’s. Professor Vermylen who performs the transplants has become an expert whose advice is sought worldwide. Secondly, having successfully completed the first marrow transplant in a child on 6 February 1974, the clinic was the first in Belgium to perform an umbilical cord blood transplant nineteen years later (25 February 1993), the successful work of Professor B. Brichard…” Extract from “La Pédiatrie” (Pedriactics) by Paul Malvaux   Furthermore, 16 years ago she went on to perform a family donor transplant on a six year-old girl suffering from the juvenile form of Krabbe’s disease. This little girl, who suffers from very few after effects, now leads a normal life.   Professor Vermylen completely understood Xavier’s mum’s dilemma. She took steps herself so that we could stay with Xavier 24 hours a day, which goes against St Luc’s normal policy. The agreement was subject to certain conditions, such as that there are three of us who take turns to stay with Xavier.   Even before they came back, an appointment was made for us to talk about performing a transplant in Belgium.   We were not prepared to let financial concerns stop us because in both cases the cost of treatment was beyond our means so we would always have been compelled to find financial support elsewhere.   Although in the short term the most important thing was saving our little angel, we also had to make sure we thought about minimising post-transplant complications, our frequent trips to the United State to monitor the illness, making the children’s isolation bearable as well as Xavier and Alexander’s quality of life.

	While he was away his little brother, aged five months, was to have his first trip to crèche but it was postponed because the night before they left his worried mum had taken him to the doctor because he was still blocked up. He had thrush, an infection in his left ear and his bronchitis hadn’t got better despite the Atrovent sprays. He was put on antibiotics (Clamoxyl).   Friday 17 December 2004. Alexander had had bronchitis and the beginning of an infection in his left ear. He had probably been infected the previous week when he was at St Luc’s (polysomnography, gastro-oesophageal ultrasound and a general check-up). The results showed that he was suffering from a considerable gastro-oesophageal reflux and slight iron deficiency anaemia. Since then he has been taking Motilium and Fer In Sol.   Xavier’s tests having been arranged at Durham and Chapel Hill, and as departure could not be postponed, his parents left with a heavy heart but comforted by the knowledge that Alexander was staying with his grandma and his godmother who would take good care of him.   Thursday 30 December. Alexander was finally better and had the last injection of his first vaccination, but the respite was short-lived.   Monday 3 January 2005. He went back to see Dr Soumoy, once again running a temperature and with a rash. She thought he might be coming down with measles but this turned out not to be the case.   Despite his state of health, Alexander was continuing to put on weight, unlike Xavier who loses weight each time he is ill. He’s a lively, smiley, sociable, inquisitive and receptive baby; he babbles away, never cries without good reason but kicks up a real fuss if there’s something he doesn’t like.

	Monday 10 January 2005. After a very tiring journey, they were happy to get home and Xavier was in great spirits; he was happy to be back in his house. To our great surprise, despite being overexcited he wasn’t sick but was really well, catching hold of his grandma for a tour of the house. He was jumping about, shrieking with happiness: phew, no change there!!   Xavier returned even more radiant and sociable because in the States he’d had his parents all to himself and apart from his visits to Duke and Chapel Hill he met lots of new faces, visited new places, discovered merry-go-rounds, etc.   In the afternoon, his mum had to do some admin (social security) so he stayed with his dad to have a long snooze to recover whilst his godmother took Alexander to the Berceau Rose et Bleu, to try for the first time the same crèche as his brother. No matter how much Sofia, Maria and Elena pampered him, it didn’t go very well because he was missing his dummy which had been left at home. The following day Alexander went back to the crèche and this time everything went smoothly; he met the other babies and had a nice day.   Wednesday 12 January 2005. Xavier and his parents went to their appointment with Professor Ch Vermylen and the first meeting was very good. It is her competence, understanding, receptiveness, compassion and her open-mindedness that made up his parents’ mind. During the interview, in which they recounted their experience in the States, she gave a few hours of her time to listen to them and answer their questions. They gave her the reports and results of tests carried out in North Carolina but she could already see that they knew more about the illness and she said that if necessary she was prepared to get in touch with Dr Kurtzberg. She also promised always to keep them informed and advised them to return to Chapel Hill regularly so Xavier’s development could be monitored.   Xavier’s parents visited the day hospital where he would be monitored following the transplant. They also went to the sterile unit which they had only seen from the outside. The unit only has six rooms, only two of which are for children. The infrastructure was very disappointing: the rooms are tiny, there’s no provision for children and even less for the parents. There is no comparison to Duke Hospital.   Although they were sure that they had made the right decision in deciding that Professor Vermylen should do the transplant, they were suddenly filled with doubts and very tempted to return to Duke because Xavier and his Mum were going to have to live in a room no bigger than a pocket handkerchief for at least eight weeks.   Friday 14 January. Xavier saw his neuropaediatrician again, who found him very well and full of life. She said that his new diet was doing him good and was happy to note that he had improved a lot since his last visit. However, she was reticent about the reports from the United States.

	Wednesday, 19 January, Alexander has a fever and his mom has to take him to his pediatrician because he is sick again with bronchitis. Dr. Soumoy prescribes respiratory physical therapy for him but no antibiotics (Neobacitracine, Altrovent spray).   After less than two weeks at the day care center, he is once again sick with his third bronchitis. He is a very frail child. Dr. Soumoy states with reason that he can no longer go to the day care center, not just for himself but also to protect his brother who is fine for the time being.   Since his return to Belgium, Xavier hasn’t gotten sick and it is important for him to be in good shape so that the transplantation doesn’t have to be postponed. The transplantation will take place once the umbilical cord blood stem cells arrive from the United States where two HLA compatible blood cords were found, but we still have to wait for the results from the additional tests that Prof. Vermylen requested: this is an advantage in comparison with Duke Hospital, where HLA compatibility was enough for the selection.   Their mom is very worried and is really feeling very low. Even if she tries her best to control her anguish, she has to make a big effort not to break down.   The next day, she decides to check if Xavier has been infected. His pediatrician reassures her that he’s in good shape but he has to be isolated from his brother.   We organize things to prevent them from being in contact but it’s not very easy because they usually share the same bedroom, even if Xavier always ends up spending the night in his parents’ bed. Alex isn’t six months old yet and he needs a lot of attention so we foresee serious difficulties. We put up gates on all the doors to prevent the two children from being together in the same room and are very strict with sanitary measures, always washing our hands before going from one child to the other.   The physical therapists that are currently coming to the house for Xavier are also doing respiratory physical therapy for Alexander who unfortunately is not getting better and always has a fever.   Wednesday, 26 January, Xavier’s mom takes him for his eye exam, which is totally reassuring and stable. When she gets home, she takes Alexander again to Dr. Soumoy who says that he should immediately see an ear, nose and throat specialist and do a blood test. The pediatrician receives these test results within two hours: Alexander has an ear infection in his right ear, a pneumococcus in his left ear and still has bronchitis. He starts taking antibiotics (Augmentin) and is prescribed Ciloxan drops for his left ear. It is now out of the question for either child to go to the day care center, and contact between the two must always be avoided at home because Xavier is scheduled to be admitted to St. Luc on Monday, 31 January.   Thursday, 27 January, despite all of our precautions, Xavier has a slight cold (Clamoxyl, Neobacitracine), and everything is postponed until the following week. We have to pay extra careful attention because time is important, and as long as he is not completely better it is impossible to begin the chemotherapy.   We also have to postpone “The Dragonflies” medical service assistance that we contacted so that during the transplantation, a pediatric nurse will come to the house to take care of Alexander three days a week and his godmother will come to take care of him the rest of the week.   Friday, 4 February, Xavier is almost completely better, and everything is scheduled again for the following Monday. We have to pay careful attention this weekend because despite the antibiotics, Alexander is sick again. His usual pediatrician is not available so his mom takes him to see Dr. Simon, who thinks that he might have pneumonia and requests an x-ray and a blood test but everything is normal. She prescribes another antibiotic (Ceclor) and he has to continue taking Ciloxan drops.   These past weeks have been very trying for all of us. Xavier often goes to the gate in front of the bedroom and doesn’t understand that he can’t go in, especially if his little brother is there. Fortunately his days are full, since besides the many times he goes to St. Luc for a variety of additional tests (neuropediatrician, dentist, ophthalmologist, logopedia…), he has his physical therapy sessions, he takes long naps with mom, and we have hired a lady who comes to play with him a couple of hours a day so that we can also take care of Alexander.   Xavier is in a period of full development and has added some words to his vocabulary: he finally says “maman” and points his finger at his mom, “o’ma” for his grandmother, “tin” for “tien” when he wants something, “wo wo” when he hears a dog bark, “br br br” when he is playing with his cars, “cou” when he plays peek-a-boo and “ch..” when he puts his finger to his lips to say “shhh.”   Before going to the U.S., he learned how to blow someone a kiss, and he now does it every time he’s happy to get his own way or when he wants to respond to us. His favorite toys are also allowed to receive kisses, and when his dad puts him to bed, Xavier says goodbye to them by waving his hand and blows them many kisses.   In Durham he offered his mom his cheek and she was able to get her first kiss, even before asking for it. Now he accepts giving kisses even if he prefers blowing them from his hand.   Xavier is starting to scribble and we buy him crayons to encourage him in this direction.

	We had decided not to have a child with IVF-PGD because we had to act as quickly as possible to save Xavier, but in the meantime we learned that there is a risk of rejection beyond the first year and a second transplantation might be necessary even if the risks are even higher. In addition, there are only 10 years of experience of cord blood cell transplantations in other cases of Krabbe’s disease, so no one can know what the future holds for Xavier.   After careful consideration we decide to go ahead with this plan after the eight weeks that Xavier will be hospitalized in a sterile room. This time we are lucky that we have two compatible donors, but it took a long time to find them, and nothing guarantees that if it we would once again need to find another donor, that we would find one on time to save Xavier, especially if his HLA is not common.   As soon as our third child is born, the blood from his or her umbilical cord will be frozen so that it will be available for Xavier throughout his life. We can directly begin the procedure because all of the steps have been taken: we did all of the preliminary analyses, we had all of the required interviews and our insurance company has given us its agreement. We only have yet to schedule an appointment with Prof. Liebaers at the AZ-VUB.   We are lucky to live in the same city as one of the highly reputed centers in this field. Unfortunately the waiting list is long but without the approval of the Ethics Committee we don’t have priority. This is why Prof. Liebaers gave us the contact information of Dr. Yuri in Chicago where the procedure is quicker.   Prof. Vermylen, who is in full agreement with us, contacts Prof. Liebaers because time is once again our enemy. We have to act very quickly but before our departure nothing was planned because the mutation had not yet been defined.   In any of the scenarios, in Belgium or in Chicago, we need the mutation to opt for this solution, and if not we will have to contemplate other solutions.

	Xavier will be hospitalised for eight weeks in a sterile room and we will have to take turns being with him. To do this we will have to wear special clothes, put on specific shoes for this unit, put on special headgear and follow the procedure for washing our hands. We will always have to wear a mask in his room, we won’t be able to use the bathroom and we won’t be able to eat or drink. His mom will be with him most of the time and his dad and his grandmother will replace her for a couple of hours every day so that she can get washed and have something to eat. His dad will also come to take over 24 hours during the weekend. We have made a schedule that we will adapt based on Xavier:     During the week:   • 7:30 a.m. to 10:30 a.m., before going to work, his dad will drop off his grandmother at St. Luc, every morning she’ll bring his pajamas, his security blankets and his blankets that have to be washed at 60° and ironed with a hot iron before individually wrapping in plastic bags that have been provided for us. She’ll give Xavier his bath and breakfast while his mom will get washed and have something to eat.   • 10:30 a.m. to 6:30 p.m. his mom will come back to stay with him. The whole time she won’t drink to avoid having to go to the bathroom which would make her leave Xavier alone for a long time, because in order to go to the bathroom she would have to leave the unit, cross the whole adjoining unit, and once back she would have to repeat the whole procedure before joining Xavier.   • 6:30 p.m. to 10:30 p.m. his dad will come to replace her so that she can go home, take a shower and have something to eat, but she also has to take care of Alexander: cuddle him, give him his evening bottle and take care of him before putting him to bed.   • 10:30 p.m. to the next morning his mom will sleep in the room with him, wearing the entire gear including the mask and putting on plastic slippers to insulate her feet.     At the weekend:   • Saturday from 2:00 p.m. to 4:00 p.m. grandma will come to take over for his mom while his parents go the cafeteria. This is the only moment during the week that they will be able to see each other without their masks.    • At 4:00 p.m. his dad will take over, and he’ll stay with him until Sunday afternoon so that his mom can go home and take care of Alexander, attend to any urgent matters and possibly have a moment to relax.   • Sunday at 4:00 p.m. his mom will come back to stay with him so that his dad will also have some time to himself in order to rest and relax before starting work again on Monday morning.     Alexander:   • From Monday to Wednesday, 7:30 a.m. to 4:30 p.m., the pediatric nurse will come to take care of Xavier’s little brother, and his grandmother will take over until the following morning.   • From Wednesday evening to Friday evening, his godmother will take care of him so that his grandmother will be free to take care of the daily matters at home.   • Saturday from 2:00 p.m. to 7:00 p.m. his godmother will come back to take care of him because his grandmother, father and mother will go to St. Luc. During the morning his grandmother will take care of Alexander; at the end of the morning his dad will go to the supermarket to do the weekly shopping and at the end of the afternoon his mom and his grandmother will take care of whatever else is necessary.   At the time being our schedule seems perfect, only time will tell if we have to make any changes to it.   Monday, 7 February:  Departure for St. Luc University Hospital. From now on we will try to give regular news about Xavier in "The transplant".   Xavier’s mom and grandma