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Saskia's BMT - Overview of the BMT

Whilst Saskia's direct family know most of the ins and outs of what's going on and where Saskia is in the process, it's proved understandably tricky for most other people to get perspective on things since the process is very complex and it takes a long time. Below is an attempt to provide an extremely simplified overview of Saskia's specific experience to provide just such perspective.

 

Overview of BMT in chronological order

2001 onwards Monthly blood transfusions for Saskia from birth, though had a year-long break trying steroid treatment which failed. Also trialled another drug which failed. From age 3, nightly night-long infusions via needle of another drug to alleviate iron-overload problems.
2003 Second child born happy and healthy. Umbilical blood collected at birth. Blood discarded later when testing reveals this child is not a tissue match / potential donor for Saskia.
2004 Mum and Dad went to Chicago for IVF / PGD. Twice. Failed both times.
2004 Tried IVF / PGD in London when it became legal for us in the UK. Pregnant.
2005 Collected blood from umbilical cord at Imogen's birth and stem cells cryogenically stored.
2006-07 BMT work-up. Many tests to make sure Saskia is suitable and healthy enough for a transplant.

Some time in March 2007 . . .

After Saskia became eligible for the transplant having passed many organ and blood tests, she had an operation (general anaesthetic) to have a Hickman Line inserted.

A few days later . . .

(25th March)

Saskia admitted to hospital and put into an isolation room. The room is kept at a pressure slightly higher than normal as clean air is pumped in continuously. People entering and leaving the room required to adhere to a strict protocol to minimise infection risk.

Saskia is given too many drugs to mention, the most notable of which are several "chemotherapy" drugs, the purpose of which is to kill off Saskia's bone marrow. The principal function of bone marrow is to make red blood cells (used to transport oxygen round the body), white blood cells (used to fight infections) and platelets (used to help the blood clot). It's the red cell production which Saskia's bone marrow does not do correctly, even though the white cell and platelet production works well. However, the production of ALL these cells will be stopped when the chemotherapy kills off her bone marrow, destroying her immune system.

10 days later . . .

(4th April)

Known as "Day Zero". The transplant day. Imogen has operation to take out some of her bone morrow, which grows back very quickly afterwards. This, together with the stem cells collected from the umbilical cord when Imogen was born in 2005, is given intravenously to Saskia. This is not an "operation" as such but a procedure similar in routine to the 50 or so blood transfusions she has had to date.

For the next few weeks loads more drugs, seemingly 24 hours by Hickman Line, nasal tube and orally. Drugs to combat the side effects of drugs. Drugs to combat the side effects of those drugs. And again. No joke. Saskia has side effects, the most obvious being all her hair falling out, being sick lots and feeling very sick.

During this time a family member was in the room with Saskia 24-hours a day.

2.5 weeks later . . .

Tests of Saskia's neutrophil count indicate that she may be starting to get the emergence of a new, though very weak immune system. Start taking short trips outside the hospital to big open spaces since Saskia is doing so well.

Moved out of big isolation room to a smaller one getting ready for going home.

A few days later . . .

Overnight home visit (we live a short walk from the hospital, so very handy). Same the next night.

2 days later . . .

(5 weeks after admission)

Discharged from hospital. We're told this is exceptionally soon after admission and that Saskia appears to have done fantastically so far.

Not able to tell what's going on in her bone marrow yet. It's too early to tell how successful the process has been in killing off her old bone marrow or whether the new bone marrow is producing red blood cells or platelets.

On 5 drugs, which is a relatively low number at this early post-discharge stage. Their purpose is: to help prevent infection by bacteria, viruses and fungi; to help prevent graft-v-host disease; and to reduce blood pressure. Getting Saskia to take these medicines is a real challenge.

Still severely immuno-deficient and at risk to infection.

Attending hospital 2-3 times a week for blood tests, check-ups and blood transfusions.

Saskia able to see friends one-on-one, and allowed to go out to wide open spaces or places with few people.

Saskia back to a less controlled diet, though some restrictions still in place.

Needs to wear sun-block outside since the process has left her skin very sensitive to the sun

Any infection could easily put her back in hospital, and we're told to "expect" readmission some time in the next 12 months for some setback or other.

Saskia is bald and beautiful.

1.5 weeks later . . .

(early/mid May)

After going in for a routine blood transfusion, Saskia is readmitted for observation and an extra intravenous antibiotic. She seems OK on whole but has been prone to occasional severe shivers which may indicate infection.

Neutrophil count still going up. Platelet count seems holding at an encouraging level, but early days.

Another blood transfusion. Certainly not making many red blood cells at the moment, which after all is the whole point.

Stops taking the drug which controls blood pressure.

She is discharged after 2 nights. No infections detected during this second stay in hospital, but doctors still extra wary. Saskia still has that cough. No shivers for a past few days.

The next week . . .

(3 weeks after first being discharged)

 

 

Saskia seems very well. She is at home but visiting the hospital 2-3 times a week. She has met up with a few friends for one-on-one play dates. Her home schooling has started: 5 hours a week is not a lot but hopefully it will keep her up to speed with her peers. There have been some minor discipline issues as would be expected from a child who, for a few months, has been spoilt for attention, been highly controlled but had no routine. The daily grind to take the many medicines is getting slightly easier but still a challenge.

Two months after first being admitted . . .(day 48 after transplant)

Results of first "chimerism" test come back to reveal how much Saskia's old bone marrow (which we hope to have killed) is still working and how much her new bone marrow is working. The test says that the bone marrow at work is at least 97% Imogen's - the test doesn't go higher than 97%.  Saskia is taking her medicines better although we are having difficulty maintaining her cyclosporin levels (that's the one that protects against Graft Vs Host Disease). We still go in to hospital 2 - 3 times a week. No more blood transfusions. Hope to see first evidence of red cell production soon.

. . .day 71 after transplant)

Second chimerism test results confirm that the only bone marrow at work is still all donor bone marrow.

Saskia's hair is affected by drugs and is barely growing back. What is there is thin, a different colour and possibly curly?? She has a lot of facial her because of one of the drugs. Saskia is put back on anti-fungal drug (voriconazole) for four weeks due to exposure to building works at the hospital (!).

Day 77 Saskia is readmitted to hospital for two days because dangerously high cyclosporin levels have damaged her kidney function (luckily reversibly). The levels have shot up because of interaction with the anti-fungal drug (voriconazole).
Day 90

Saskia needs daily blood tests to establish the correct levels of cyclosporin while she is on the voriconazole.  Third chimerism test done.

(100 days after transplant) Saskia is now at a decreased risk of one particular virus occurring. We should also get back the third set of chimerism test results. If they show 90% or above of the marow production comes from the donor, we should be able to start weaning Saskia off the cyclosporin 4.5 months after transplant (mid August). If Saskia had not have been put back on an anti-fungal drug we would have taken the Hickman line out about now.
Day 106 Saskia comes off the anti-fungal drug (voriconazole) and will need regular blood tests to establish the correct dose of cyclosporin.
Mid July

Saskia readmitted to hospital with a high temperature and general symptoms of an infection. She receives various intravenous antibiotics.

This is the 3rd time she has been readmitted to hospital since her main spell there for the Transplant itself. Saskia's  Hickman Line is removed when the infection cannot be shifted with antibiotics. Saskia's red blood cell count drops a little (we hope because of her medications/infection). Third chimerism result says that the bone marrow at work is at least 95% Imogen's (most likely all of it). Saskia is discharged after 9 days.

Late July - early August.

 

Saskia is still on 4 different drugs. We await the passage of time to start weaning off some of these drugs. She is still bald, but a fuzziness is developing.

128 days after transplant (approximately 4.5 months) Saskia showns no signs of graft-v-host disease (GVHD) and the fourth set of chimerism test results are good. Saskia is weaned off the drug used to help prevent rejection of the new marrow. This weaning takes 6 weeks. Saskia does not develop GVHD or reject the bone marrow.

Saskia's hair is now starting to make a determined reappearance.

176 days (6 months) after transplant

Saskia returns to school. This is it: The Major Milestone.

She will be slightly more prone to infection than a normal person for the next 6 months, but well enough to participate in normal life in every way.

In 2 weeks time we hope to be able to discontinue 2 of the remaining 3 drugs Saskia is on (nearly) daily.

October 2007 Saskia is settling back into school as if nothing has happened. Her hair is growing back and in a few months will simply look a little short. She's on penicillin twice a day, but no other medicines. Her 6-weekly checkups in the hospital will be cut down in the New Year, and we look forward to next Spring when we can give her all the normal immunity jabs kids get.
January 2009 Saskia is progressing wonderfully. Mum and Dad are very proud of her. She takes penicillin orally twice a day, and this will persist indefinitely. Once a month, and only for a few more months, she has a venosection: removing blood to help reduce the iron concentration in her body which built up from those 50+ transfusions she's had through her life. She's doing well at school and well up with her peers. She's very happy. She's also very active, and got 3rd place in her class in last summer's sports day! And her little sisters and little brother are doing very well too.

The future

Semi-annual check-ups and then annual check-ups for life.

On penicillin for life (or possibly only till adulthood).

First person on Mars / President of Atlantis / Inventor of World Peace / Happy and Healthy Person.

 

Please, if you're reading this as a patient (or their family) bear in mind that this is a very superficial description. Also, it only relates to Saskia's particular condition and to the particular treatment she has received at one particular hospital given the particular way she happens to have responded. If you've heard things should be done differently it does not mean that there is necessarily a contradiction. Certainly, Saskia was fortunate and did relatively well in terms of how she has felt during the process and how quickly she came through it. Please don't try to stretch comparisons with your case.