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Newsletter, Issue #4


MAY NEWSLETTER
Welcome to the 4th newsletter of www.naitbabies.org

In This Issue


NAITBABIES ON SOUNDING BOARD for Prophylix Pharma AS
http://www.prophylixpharma.com
Interview with Dr Jens Kjeldsen-Kragh MD PhD
(Becky Robinson)
 
Dr Jens Kjeldsen-KraghDr Jens Kjeldsen-Kragh
We are excited to announce that Naitbabies have been asked to join the Sounding Board for Prophylix Pharma AS (PPAS) which also includes Professor Dick Oepkes, Professor Stanislaw Urbaniak and Professor James B Bussell.  PROFNAIT is a collaborative project aiming to develop the first drug that will be able to prevent severe bleeding in foetuses and newborns due to foetal/neonatal alloimmune thrombocytopenia (FNAIT). The project will establish the scientific, clinical and quality documentation that is required to obtain Marketing Authorization in the EU for the new drug. In addition to Prophylix the project includes 8 consortium partners within Norway, Sweden, Denmark and Germany.

Dr. Jens Kjeldsen-Kragh is the Chief Scientific Officer at Prophylix Pharma, and he was kind enough to take some time out of his very busy schedule to talk to me about himself, his work and the very exciting PROFNAIT project.

Dr. Kjeldsen-Kragh is a specialist in Immunology and Transfusion Medicine and has been involved in research on blood platelets for more than a decade. In 2000 Dr. Kjeldsen-Kragh and colleagues at the University Hospital of North Norway in Tromsø planned a very large prospective observational trial of HPA 1a-negative women – the largest prospective study of FNAIT ever performed.  More than 100,000 pregnant women were HPA 1 typed, and those who were HPA 1a-negative (2.1%) were screened for anti-HPA 1a (10.6% of these).  The pregnancies of the HPA 1a-negative women were followed and the mother’s antibody levels were tested at regular intervals.  The study found that the majority of women are immunised against HPA-1a after delivery of their baby.  This is similar to the situation that occurs in haemolytic disease of the newborn (HDN) where a RhD negative woman gives birth to a RhD positive child. With HDN, during birth, the mother may be exposed to the infant's blood, and this causes the development of antibodies, which may affect the health of an RhD positive foetus in subsequent pregnancies. Today RhD negative mothers who have given birth to an RhD positive child are given an injection of anti-RhD immunoglobulin (commonly known as anti-D) to reduce the risk of any subsequent babies being affected by maternal antibodies against RhD.  Dr. Kjeldsen-Kragh and his collaborators in Tromsø began researching the possibility of using the same approach against HPA-1a.

Together with researchers in Toronto, Canada, the Norwegian team made a proof-of-concept study in a mouse model of FNAIT.  The study proved that, conceptually, a prophylactic administration of platelet antibodies leads to suppression of antibody production and prevents poor pregnancy outcome in FNAIT. (A prophylactic drug is one that is used to prevent a disease, rather than to treat or cure a disease).
 
The next stage in the process, following on from the mouse study, was to develop a prophylactic drug to be used on humans.  As FNAIT is classified as a rare condition, Prophylix Pharma was granted ‘Orphan Drug Designation’ for their prophylactic drug by the European Medicines Agency.  Benefits and privileges are associated with Orphan Drug Designation and is used to encourage pharmaceutical companies to develop medicinal products for rare conditions, as otherwise it would not be cost effective for companies to do so.  Obtaining this designation was a huge breakthrough and meant it was possible for Prophylix Pharma to apply to the EU Commission for a grant of €6m in order to develop the drug.  They received a very favourable evaluation from the commission and are currently in the final negotiations to obtain the grant.
 
PPAS along with the consortium members are now in a position to begin the massive task of collecting blood plasma from hundreds of women who have antibodies against HPA-1a.  Immunoglobulin G needs to be extracted from the donated blood plasma and purified to produce the drug. 2,000 litres of blood plasma is required at this stage of the procedure.  Once enough plasma has been collected, the human testing (phases I and II) will begin.  This will take place at the University of Tromsø.  The purpose of the phase I/II trial is to determine the level of dosage that will be used and to assess if there is any toxicity associated with the drug.  Dr Kjeldsen-Kragh stated they don’t believe there will be any toxicity issues as the drug is so similar to that used for HDN – the only difference being the specificity of the antibody used in the drug – in this case platelet antibodies.  This stage of trials is required for all medications.
 
The final phase (phase III) of the testing will involve screening 75,000 pregnant women to ascertain their platelet type.  From this sample of 75,000 it is expected that 2% will be HPA 1a-negative. Those that have not developed antibodies during the pregnancy will be used in the trial. These approximately 1,500 women will be split into two groups.  Shortly after giving birth to an HPA 1a-positive child, one group will receive the prophylactic treatment, and the other group will receive a placebo.  This will be a double blind study, so neither the women participating in the trial, nor the researchers will know who has been administered with the prophylactic.  All of the mothers will be tested several times during a year after delivery to establish whether antibodies have developed following giving birth.  The results from both of the groups will be tested and compared.  If there is a significant difference in the two groups then this will be formal proof that the drug is effective.  Once all the relevant paperwork and approvals have gone through following the trials, then the drug can go to market. 
 
The donation and collection of blood plasma is a big challenge for the team.  They need as many women as possible who have antibodies against HPA-1a to sign up and register their interest in assisting the project.  While the project is based in Europe, Dr Kjeldsen-Kragh has asked for women all over the world to sign up and register their information so they can be contacted should the collection of plasma be required from elsewhere. 

Plasma is collected by a special procedure called plasmapheresis. Blood is collected the same way as in a normal blood donation, but during plasmapheresis a special piece of equipment - the apheresis machine – is used to separate plasma from the blood by centrifugation while returning the blood cells (red cells, white cells and blood platelets) to the donor. In other words, you will keep all of your valuable blood cells and only the plasma will be collected. It is usual to collect around 600 ml plasma in one procedure which takes from 30 to 60 min.  Donors can safely give blood plasma one or two times a month. 
 
If you would like to help, please go to http://www.prophylixpharma.com/donate-plasma/.


NAIT MOMS FROM USA & CANADA MEET UP IN CHICAGO
(Marin MacCallum)

On March 23 a group of NAIT Moms from USA and Canada finally had a chance to meet in Chicago, Illinois.
Angie explained, “I had spent the last 7yrs of my life chatting and sharing life details with some of these women and meeting them in person solidified the bonds we had.  I had wondered if our common interests and bonds would reach beyond NAIT and they have surpassed any expectations I could have ever had.  We connect in more ways through family and relationships and parenting - some of the best relationships in my life right now”.  Stephanie is the founder of the NAIT Yahoo group which she started 10 years ago and said, “I needed to meet because NAIT is so lonely. No one can really understand what it is like to have your body attack your baby and there isn't a cure. RhD is similar but that has a cure.  We are so rare and that makes it lonely.” Erin found this group of friendship and support a year and a half ago, “I wanted to meet the women who grant me strength. These are the women that have shared my highest levels of joy and the purest terror of NAIT.”
 
The ladies spent 3 nights together in a luxurious condo.  Angie described it as a “Sleek, modern and spacious condo on the 38th floor with fantastic views”.  Stephanie said “Angela found an amazing place; we all got to act like millionaires”.  Angie recalled “The whole visit was like a grown-up slumber party that lasted an entire weekend.  We did all the things girls do together - laughing, crying, and talking late into the night”.  Georgena added “It amazed me that seven gals who had never met, felt like we were with best friends and no weirdness. Girlfriends are the best!!”.
While in Chicago the ladies had a chance to tour the city.  “We ate, drank, cried, laughed, drank & ate.  A little shopping and site seeing too.” said Angie and Erin. “It is an awesome city!” They also stopped at Build a Bear at Navy Pier. “We literally put our hearts into it! Each heart had our kids’ names on it so it is filled with lots of love”.
 
This was definitely a weekend to remember and the ladies are looking forward to meeting again.
Shelley's favourite part of the weekend was “finally being able to personally meet these wonderful women who are such a big part of everyone’s lives. I can now hear their lovely voices and hear their demeanour when I read their posts. I love it!”  Stephanie said “It wasn't who knew what more or who had it better or worse, we were all just there for each other.”  One of Erin's favourite parts of the weekend was “getting to hear more about everyone's kids. Not just the NAIT effects or treatments, the funny kid stories.”
 
Stephanie shares what she has learned from NAIT, “I learned that out of tragedy comes good... the good that came was obviously Chris and Jason my miracles but also the love from everyone else who deals with this. I learned that even online you can build friendships that are true.  When I started yahoo in 2002 people still looked at meeting online as being a freak.  The mention of speaking to people who you didn’t know would get comments and eye rolls.  But this has taught me that I can reach out for help and it is there.  That good people help and no matter how hard this has been that the power of the group is so unbelievably strong.  I also am shocked that I want to do this again and again.  I miss what we shared and can not wait for the next NAIT trip.”
Georgina quotes the words from her daughter Ruthie “My stranger friends are now my real ones".Angie Dugan, Katherine Taylor-Jones, Shelley Staples-Marfleet, Erin Hickey, Janene Melendez, Stephanie Volpe(Angie Dugan, Katherine Taylor-Jones, Shelley Staples-Marfleet, Erin Hickey, Janene Melendez, Stephanie Volpe)
Shelley, Stephanie, Erin, Georgena Zulpan, Angie(Shelley, Stephanie, Erin, Georgena Zulpan, Angie)


PRACTICE DAY FOR 3 PEAKS CHALLENGE
(Becky Robinson)
 
On 22nd April 2012 a bunch of intrepid climbers put on their walking boots and woolly hats to brave the rain, hail, wind, sleet and snow to climb to the top of Mount Snowdon - an altitude of 1,085 metres (3,560 feet) and the highest mountain in Wales.  This was a training climb to help prepare everyone for the 3 Peaks Challenge later in the year. 
 
The group hadn’t all met each other before the big day, but it wasn’t long before they all felt like old friends. Wearing matching NAIT awareness t-shirts, they managed to reach the top in about 3½ hours which was a great achievement considering the unfavourable weather conditions, the fact that there was probably a lot of gossiping going on, and that one of the party had a broken arm!  Thea Palmer unfortunately slipped over whilst in training for the training climb and ended up in a plaster cast, but this didn’t stop her completing the climb (though it did stop her from cutting up her food at dinner during the evening before and Nia Llwyd Lewis had to assist her!)
It wasn’t an easy climb and there was a scary moment for Andrea when her boots slid in the icy snow on a steep stretch near the top!  Fortunately she regained her footing and everyone made it to the summit in one piece. The climb down took another 3 ½ hours and the climbers were able to take in the breathtaking and beautiful scenery.
The climb has given everyone a confidence boost, and a feel of what the 3 Peaks Challenge will be like.   Thea commented after the climb that “Best of all was meeting up with peple who I had only spoken to via the internet, the sense of real exhilaration and achievement at climbing Snowdon whilst spreading more awareness about NAIT!”
A huge well done to all the climbers: Thea Palmer, Stacy Corke, Andrea Palmer, Lee J Palmer, Gary Gill, Tracie Gill, Nicole Gill, Micky Gill, Dwayne Gill, Kylie Cooper, Delphine Coleman, Marcus Davies-Friend, Clare Everitt, Steven Fisher, John Saunders and David Andrews.And thank you to the support team: Victoria Davies-Friend and children, Nia Llwyd Lewis and her children, along with Senen and John Paul Rivett were all part of the support team offering encouragement.
Roll on July when the real challenge begins...
SnowdonSnowdon


MEMBER SPOTLIGHT
(Becky Robinson)

Vikki Dan and TianaVikki Dan and Tiana
Vikki Plum Brant lives in Devon, England with her husband Dan and her gorgeous daughter Tiana.  Vikki first found out about NAIT whilst pregnant with Tiana.  During a routine check-up when she was 25 weeks pregnant Tiana's heart rate was higher than normal.  She was an active baby, so following a check 20 minutes later Vikki was sent home.  At her 28 week check, her baby's heart rate again was high, so Vikki was sent to the hospital to have an ECG and get a more thorough assessment of the baby's heart-rate. After four days of monitoring, the heart rate of the baby was still high and Vikki was offered a scan.  It was at this scan that what looked like a small bleed was detected in Tiana' s brain.  At first, it wasn't clear if what was showing was a bleed, a tumour, or the result of an infection. Vikki was immediately referred to a consultant and had to sit and wait while the nurses found the consultant to come and speak to her. 
 
The consultant reviewed the scan and concluded that it was most likely a bleed that was caused by an infection such as toxoplasmosis.   Blood tests were taken, but they all came back clear of infection.  Her consultant then liaised with the foetal medicine unit at Bristol St. Michaels who thought that the only likely thing to cause a bleed would be NAIT. It took two weeks before Vikki and her partner were given a definite diagnosis.  It was NAIT, and so a trip to Bristol followed so that Vikki could talk through possible options with the specialist.  Vikki was told about the condition and the risks to her baby.  She was informed that platelets could be ordered and she would be able to return the following week so that the baby could receive a platelet transfusion. At the same time as having this transfusion, a FBS (foetal blood sample) would be taken.  The FBS carried a 5% risk that Vikki's baby would die. Before the procedure, Vikki had another scan which revealed the extent of Tiana's bleed – it was severe.  Vikki was asked if she wanted to go through with the planned procedure, or if she wanted to terminate the pregnancy as the bleed was so extensive.  Vikki was adamant that she was going to carry on with the pregnancy which by this stage was at 33 weeks.  Vikki was unsure about having the FBS because of the risks involved but her consultant advised her that her baby would more than likely not survive labour without the platelet transfusion. 
 
On 17th June 2005, Vikki went to Bristol for the FBS procedure.  She went first to one hospital for an MRI to again determine the extent of the bleeding, and then onto St. Michael's for the FBS.  Tiana's platelet count was 7k.  The transfusion got her levels up to 121k.  Vikki was booked in to deliver her baby 3 days later.
 
Vikki was warned that the damage to her daughter's brain was likely to be so severe that it was probable that she would have to spend her life in an institution.
 
Tiana was delivered at 33 weeks and 3 days.  Her platelet counts were 88k and she was one of the healthiest babies in the special care baby unit!  She had a platelet transfusion, and when she was 2 days old her platelet count started to creep up.  Other than slight jaundice Tiana was well enough to be allowed home at 10 days old.
 Vikki Dan and TianaVikki Dan and Tiana
Tiana is now almost 7 years old.  She has been diagnosed with mild cerebral palsy and as a result can't run as fast as some of her friends and sometimes struggles with fine motor skills.  She wears a splint on her left hand and foot.  She has a visual impairment - she has no peripheral vision and nystagmus which means she needs to hold her head at a certain angle when concentrating.  She needs print slightly larger to be able to read it.  However, she is coping so well, and she never lets anything bother her and, as Vikki says, “To me, she’s perfect”.
 
Vikki started to try and set up a support group for NAIT, and met Nia Llwyd Lewis who was pregnant and also looking for support.  Nia found the Facebook support group and both her and Vikki joined.  It was through meeting Nia and joining the group that Vikki began to change her mind about never getting pregnant again which had been her reaction following Tiana's pregnancy.  Vikki up until that point had been unaware that there was treatment that could prevent the same things that happened to Tiana from happening again. 
When Tiana was 12 months old, Vikki split from her father.  She met Dan a couple of months later and he became Tiana's daddy.  Vikki was open with Dan from the beginning that she would never be having any more children and he was fine with that at first, but as time went on it became something that they both dreamed of.  When Vikki and Dan married in 2009 they had blood tests done to see if they would be affected.  They were in the 100% group.  Vikki threw herself into becoming qualified and establishing a career as a hairdresser, but all the time researching more about NAIT and visiting the group a lot more, asking questions. 
 
In September 2011, Vikki, Dan and Tiana moved house and Vikki felt that the time was now right to try for another baby knowing full well what the pregnancy would involve.  On 22nd December 2011, Vikki had a positive pregnancy test!
 
Vikki had scans at 10 weeks, and 12 weeks, and began weekly 1g/kg IVIG treatment at 14 weeks.  Following the first treatment she experienced severe migraines after 72 hours, but this improved in the following weeks as the administering of the IVIG was slowed down.  At 19 weeks, Vikki started to experience visual disturbances and loss of energy which was very difficult for her to cope with.  At 23 weeks, Vikki was prescribed Prednisone, a steroid that would help with the side effects that she was experiencing, and also help to protect her baby. This has helped and Vikki is now able to function normally.  Her visual disturbances have decreased dramatically and she is full of energy.  She can now do the normal things in life like take Tiana to school and do the shopping.  Tiana, incidentally, is extremely excited about being a big sister – she has even sorted through her toys and books to make some room ready for her little sister and has got rid of all her little bits that "the baby may choke on" (Tiana’s words!).
 
Vikki is still undecided about having an FBS this time round.  She will be having scans every 2 weeks for the duration of her pregnancy and wants to deliver at 34 weeks.
 
We wish Vikki, Dan and Tiana all the best for the rest of the pregnancy and we are looking forward to hearing about their new arrival very soon!





Upcoming Events

MAY
19TH


Weybridge UK,  "pampered chef" demonstration, a clothes show, cupcakes, and jewellery.

JULY
21ST 22ND


3 Peaks Challenge (Scotland/England/Wales)



ABOUT NAITBABIES

Naitbabies.org is an organization that includes families from all over the world who have been affected by NAIT.  We communicate with each other daily as we offer friendship and support. We hope for more research, new treatment options and prenatal screening for all women.  We are raising awareness of this disease in many ways. We keep updated on the most recent research.  We have included many articles to keep you informed on what is happening within our organization.


BIRTH ANNOUNCEMENTS

Ben Andrew Parsons
Born: March 28th
To Corey & Jason Parsons
Halifax, Nova Scotia, Canada
Treatment - (week 7-27) 70g/week IVIG, (week 28-34) 140g/week IVIG 
Platelet count at birth - 324,000
Planned C-Section at 37 weeks, but an emergency section was needed at 34 weeks due to mother’s illness (HELLP Syndrome).
Ben Parsons
Marlie-May McKessar
Born: March 6th
To Kirsten and Luke McKessar
Platelet count of 112k from cord
Treatment - IVIG x 2 weekly no prednisone, no PUBS
Marlie-Mae had 3mls of Immunoglobulin at birth and platelets increased to 550k!!! Marlie Mae
Chloe Belle Von Herbulis
Born: January 11th, at 9:36am; weight 6lbs 13oz; Length 20 1/2 in, via c-section at 37 weeks 4 days.
To Donna and Eric Von Herbulis
Treatment – IVIG once a week at 18 weeks, 1g/kg, no pubs, no prednisone.
Chloe’s platelet count was 38K at birth. She received one IVIG infusion and her count rose to 70K by the next morning.   After that counts rose continually without any further treatments. Chloe Belle
Elyse Lillian Henshall
Born: February 24th
To Jennifer & Taylor Henshall
Big sister: Clara
Treatment -1gm/kg of IVIG twice per week from 20 weeks, added 30mg per day of prednisone from 32-36 weeks and had a c-section at 36 weeks, 4 days. Elyse lillian


AWARENESS / FUND RAISING INITIATIVES

(Marin MacCallum)
 
Spring Break Party – Cornwall, UK
Andrea Palmer, Jon-Paul Rivett and friend Andrew Ferguson organized an event at the Salt Bar, Hayle on April 7th. They were very pleased with the evening.  Andrea said “There was an amazing turn out for the event.  I estimate that there were at least 200 people who came to show their support.  The night was fun with every one having a dance and a laugh.  People seemed really interested in learning about NAIT and spreading the word.  The raffle and auction went super well.  We managed to raise £690!!  We never imagined we would get that close!!!! The feedback was great, after the event people were saying they had a fab time and they can't wait for the next one.  This will be in September; we hope to raise money for specialist sensory equipment for our daughter Senen as well as naitbabies.  We were so pleased with how the night went, and at how many people, friends and strangers, showed their support.  They were genuinely interested in learning about NAIT, and the money we raised exceeded expectations. A very successful night!” Salt barSalt bar
 
St. David’s Day Celebration and NAIT Awareness – Wales, UK
Nia Llwyd Lewis organized an event on Thursday, 1st March 2012, to celebrate St. David’s Day, the feast day of Saint David the patron saint of Wales, and to raise awareness and funds for NAIT.  “I decided to sell a mountain of cakes and knitted daffodils; the daffodil is one of the emblems of Wales and is traditionally worn on St. David’s Day, and some quizzes at work.  I sold 66 quiz forms, 90 daffodils and a massive amount of cakes! My friend’s sister kindly made 13 boxes of lovely cupcakes for me to sell.
The amount raised came to £250 which I’m really pleased about as I didn’t think I’d raise half that amount!!” Nia's childrenNia's children
NiaNia's cakes
 
Maureen Gill’s Jewellery and Gift Sale – Republic of Ireland
The Shamrock, Falcarragh, Co Donegal.
Thank you to the lovely people of Donegal for supporting Louise and Maureen Gill's NAIT awareness gift and jewellery and sale. Raising a grand total of £205!! (€250) (picture #10a or 10b)Maureen Gillawareness gift and jewellery
Maureen with DanielMaureen with Daniel


NAIT AWARENESS BEARS

NAIT Bear had a visit with Nikki Ribbon and her family in Corndale, NSW, Australia.
 Nikki said “He spent hours with the kids in our own front yard, checking out Thomas the rabbit, riding Toffee and Liz the goats, and Baza the dog”. He also “got licked by a wombat, met a hawk, a snake, a cockatoo, a kangaroo, even an emu”. NAIT Bear saw beautiful waterfalls and visited many places with the family. Please visit his blog to read more and see the wonderful pictures from his visit.
http://www.blog.naitbabies.org/Nait in AustraliaNait in AustraliaNait and cat puppet-AustraliaNait and cat puppet-Australia



UPCOMING EVENTS

Gary Gill – 3 Peaks Challenge 21-22 July 2012 (Scotland /England /Wales)
This team event will take place over 24hrs climbing Ben Nevis in the Scottish Highlands, Scafell Pike in the English Lake District, and Snowdon in the Snowdonia National Park, north Wales.
 If you require more information please contact us info@naitbabies.org
John Saunders and Mickey GillJohn Saunders and Mickey Gill

Saturday 19th May - Weybridge UK
Stacy Corke and her sister Denia Johnson are holding a fund raising evening including a "pampered chef" demonstration, a clothes show, cupcakes, and jewellery.






Newsletter Team

Editor/ Reporter – Marin MacCallum
Reporter – Louise Gill
Reporter – Becky  Robinson
Newsletter draft development – Stacy Corke, Thea Palmer, Jennifer Mathieu Henshall
 
Thank you to everyone who provided information for our May Newsletter. 
Please contact us with any comments or suggestions for future newsletters.
marin-newsletter@naitbabies.org


Disclaimer - Information in this newsletter is for the express purpose of raising awareness and does not necessarily reflect the views of Naitbabies.org. All medical information should be reviewed with your doctor before being acted upon.








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