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Long shot, but does anyone here have a child with Neurofibromatosis?

60 replies

Dustyblue · 10/01/2022 08:19

Hello all, asking from Australia.

DS5 was diagnosed last year (via genetic screen and paed neurologist) with NF1. The assessment process is very long. We're waiting on more genetic assays to be done for us parents and DS again, plus a learning assessment. He's seen a paed opthamologist, and no optic gliomas were detected, so that's good.

Meanwhile, he's been seeing an OT, a Physio and a Podiatrist about his various issues. He starts primary school in Feb; I won't detail all my fears about that.

There are support groups online, but the main ones I've found have been run by people who have NF themselves. Now, there's a wealth of info there, but also lots of unhelpful things such as:

  • I didn't know I had NF until I was 44, when surgeons spent 12 hours removing a tumour wrapped around my heart (Cheers, taa for that)
  • I have NF but my children don't, you need to stick to the following diet plan to stop him developing tumours (Ahem, ok)

I'm a tad worn down with it all. Any experience here at all?

Thanks in advance

OP posts:
Dustyblue · 10/01/2022 08:20

Sorry- meant to say I did a search here but there wasn't much and nothing recent.

Cheers.

OP posts:
Itonlytakesonetree · 10/01/2022 08:25

I'm not a parent of a child with NF but I do work with children who have it. In school it tends to present with difficulties with emotional regulation and learning. Language can sometimes be an issue also. We often see children getting upset quickly and often becoming very hot and sweaty and struggling to calm down. In terms of learning, children often need lots of small steps and repetition. As children go further through the school, they sometimes need a higher level of support. Language can sometimes be unclear and children need processing time.
Apologies if that was not remotely helpful, but at least it bumps it for you!

Dustyblue · 11/01/2022 09:54

Thanks for replying!

NF is supposed to be 1 in 2500 - 3000 people in Australia, which you wouldn't call rare, but you wouldn't know it when info-seeking.

Your post was actually very helpful- I can see much of DS in what you say you've experienced. It has such a vast range of effects though. DS has decent language, but can't write or draw. His reading is well above average, but he's terribly clumsy and has attention issues. Gaah, there's almost something new weekly- dreading his start of primary school.

The hot and sweaty thing is interesting. We've always noticed that he 'runs hot' & I'd not considered it might be part of NF.

Anyway, since it looks like this thread will die now- just wanted to say thanks Smile

OP posts:

Interested in this thread?

Then you might like threads about these subjects:

Rachaelrachael · 11/01/2022 10:08

Hi, my baby is currently awaiting test results as she has around 10 cafe au lait marks on her body. The Dr we saw said its 'moderately likely' that she has NF1 or legius syndrome..
She has already been through open heart surgery at 5 months old (completely unrelated) so we're devastated.
I have found the same that there's lots of stories from adults but not much information on what to expect if your child is diagnosed. I've actually stopped myself googling as some of the stuff online is terrifying. Do you mind me asking what the 1st signs of NF1 were in your DS?
Sorry I cant answer your questions, just wanted to say we're in the same boat and I hope you can find some answers

horseymum · 11/01/2022 10:23

I have a family member with it, he is not really affected at all so far and he's a young teen. It's so unpredictable I think that is the stressful thing for his mum. I also worked with a student who had it, lots of benign tumours but more making her self conscious than affecting her physically. She also had a learning disability. I know a family near us and the dad had it and had to have his leg amputated due to tumours. No learning disabilities. He reckons his mum had it but was never diagnosed. One of their children had it too but no real impact on him yet. It's so variable. I hope you find some further support from parents who are living with this, I think the stress of not knowing is hard so look after yourself.

Dustyblue · 12/01/2022 05:39

@Rachaelrachael

Hi, my baby is currently awaiting test results as she has around 10 cafe au lait marks on her body. The Dr we saw said its 'moderately likely' that she has NF1 or legius syndrome.. She has already been through open heart surgery at 5 months old (completely unrelated) so we're devastated. I have found the same that there's lots of stories from adults but not much information on what to expect if your child is diagnosed. I've actually stopped myself googling as some of the stuff online is terrifying. Do you mind me asking what the 1st signs of NF1 were in your DS? Sorry I cant answer your questions, just wanted to say we're in the same boat and I hope you can find some answers
I'm sorry you're going through the testing/waiting process. Especially after your DD having heart surgery so young.

For DS the markers were the 10+ cafe au laits, large head (we were struggling to find kids hats to fit, although he doesn't look out of proportion) and some kind of learning disability.... he had trouble paying attention and following instruction at kinder. Paed was suspicious and did the gene test, and here we are.

I wish you all the luck in the world that your test comes back clear. If not, take heart that the range of presentation is so vast- yes it's driving me nuts but that's way better than some of the disastrous conditions a kid could have.

If it does come back detecting NF1, we may have to start our own parents group!

OP posts:
seekinglondonlife · 12/01/2022 06:21

Someone I closely work with has it, as does her 20 something year old son. The mum definitely has issues that I assumed were maybe ASD - she has meltdown type things where she just can't seem to cope with new things or too much info too soon. She's very articulate though and no learning disabilities. The son seems to be largely unaffected, I only know he has it because she told me. He doesn't have any neurofibromas whereas the mum has a lot on her face and arms.

Dustyblue · 12/01/2022 06:50

@seekinglondonlife

Someone I closely work with has it, as does her 20 something year old son. The mum definitely has issues that I assumed were maybe ASD - she has meltdown type things where she just can't seem to cope with new things or too much info too soon. She's very articulate though and no learning disabilities. The son seems to be largely unaffected, I only know he has it because she told me. He doesn't have any neurofibromas whereas the mum has a lot on her face and arms.
Thanks for replying. Your anecdote demonstrates how differently people can be affected.

DS also has meltdown type things like you describe in your colleague. Then again, he's 5. He's been 'cleared' of ASD by the Paed, but it seems NF results in various processing abilities. Maybe some persist through adulthood.

Sigh. This is a tricky business to navigate for your child without panicking.

OP posts:
Dustyblue · 12/01/2022 07:24

@horseymum

I have a family member with it, he is not really affected at all so far and he's a young teen. It's so unpredictable I think that is the stressful thing for his mum. I also worked with a student who had it, lots of benign tumours but more making her self conscious than affecting her physically. She also had a learning disability. I know a family near us and the dad had it and had to have his leg amputated due to tumours. No learning disabilities. He reckons his mum had it but was never diagnosed. One of their children had it too but no real impact on him yet. It's so variable. I hope you find some further support from parents who are living with this, I think the stress of not knowing is hard so look after yourself.
Appreciate your reply, thanks. Unpredictable is right! Although as I said upthread, I guess that's better than being given flat-out bad news with no hope.

DS has no fibromas yet, and it's very hard to stop myself lump-spotting. Interestingly, one of his current issues is a 3.5cm discrepancy in his tibia (lower leg) bone. Left is longer and causing his foot to rotate & causing 'excessive pronation', whatever that means.

I don't think it's tumour/fibroma related though. NF is also known to cause early growth. He's seeing an Orthopedic Surgeon for assessment at some point- I will try to erase the word 'amputation' from my brain!

OP posts:
NFmum · 12/01/2022 08:02

I have one DC with NF, who is an adult.

Do you have any NF clinics/specialist nurses that you could access? In the UK we have these in a couple of hospitals, and they can really help support families with their questions. There is also this online support group that has a helpline, but it is UK based nervetumours.org.uk/ - try not to worry about the name, it isn't very helpful I know! I don't know if there are similar ones in Australia?

As you've probably seen from the above comments and online discussions NF is a really varied condition, and so each person's journey can be really individual. I know quite a lot of people with NF: some barely knowing they have the condition and just having a few cafe-au-lait marks and leading normal adult lives, and a few people with a lot more issues, so it can be really hard if you have a young child to know how they will be affected. It can also be hard to look online as it is mostly used by people with more complex issues, which can be overwhelming at the start. As your child gets older, some of the rarer conditions become less likely, which is reassuring. Fibromas mostly start appearing in the teenage/adult years, my DC only has one tiny one so far and is an adult.

There are some learning difficulties that can affect some children (again not all children experience these), difficulties with maths etc. If your child is finding it difficult to write, then it might be a good idea to introduce a laptop early on. My DC used a laptop from Reception as they couldn't write, and did all their school work on it.

The initial stages after diagnosis with NF can be very stressful, especially with the unpredictability of it, but a lot of people do have positive outcomes it's a pity there aren't more stories of those online!

Dustyblue · 12/01/2022 08:43

@NFmum

Thank you so much. I got a little weepy reading your post.

I'll reply properly later tonight. It's 7.30pm here and I'm late with dinner after too much MN Blush

OP posts:
Whitefire · 12/01/2022 08:53

I have NF as does one of my dc. We have a very strong family link (my Dad also has it)

I will come back and post more.

Whitefire · 12/01/2022 22:06

Sorry I have taken all day to come back.

Our family link is strong. I have 3 children, ds (the middle one) has it. I am one of three as well and only I have it. (I always joke that Dad can never deny I'm his.)

Dad was one of 5, it is likely it has come down his Mum's side. One sister doesn't have it, one sister does, both his brothers died before the family were alerted to NF but one of them definitely had it, the other is unknown.

Then my Aunt - 2 of her 4 have NF, I think with my Uncle it is just the one. Then my cousins children have NF as well.

So I have had very little problems - cafe-au-laits and starting to get some fibromas (I'm 43) DS has a massive head (I was very pleased he came at 36 weeks), cafe-au-laits and he does look like he is starting to have 'lumps'. He also has a diagnosis of ASD. (He is almost 14)

My Dad has a lot of fibromas, he has had to have some removed due to there location.

In terms of my cousins and their families, it has been varied. My one cousin has had to have fibromas removed from the sole of his foot, a cousin's child has had to have various surgeries due to bone growth issues and another has a very large plexiform on her back. So even within a family it can vary greatly.

For me, both as a NF person and as a parent it has perhaps been an easier journey because NF has always been part of my life, my sister's and I were very young when it was first picked up in my cousin and they advised for the whole family to be tested. It then meant that my children were followed through from a very young age by the regional genetics unit.

It is actually his autism that causes us the most difficulties. NF wise he sees the consultant once a year and he has now been discharged from the eye department.

Cocolapew · 12/01/2022 23:00

My DH and DD1 (24) both have it. DHs back is completely covered with lumps and a few years ago they started to appear on his neck and face.
DD has some smallish lumps on her back and breasts. She has a slightly larger head and us just over 5 ft. She was was very slow to talk, ride a bike etc. She has anxiety and presents with ASD traits.
She was very academic and had no learning difficulties.

Dustyblue · 13/01/2022 05:55

Oh wow, thanks so much for these replies.

I'm feeling a bit overwhelmed, and to be honest a bit frightened BUT I did ask for experiences, and I'm grateful you all took the time to reply.

@Whitefire, that sure is some decent family history. We still don't know if this is inherited, or a spontaneous mutation. Apparently with NF, the stats are currently running at close to 50/50 (so of people with NF, 50% inherited it from a parent & 50% have a spontaneous mutation at conception).

Still waiting on those tests to be done. There's no known history but DH thinks it might be his side (for various reasons). He has a few odd lumps himself, but no other markers. I think it could just as easily be a spontaneous mutation caused by an old egg (I was 41.5 when I had him).

@NFmum, I think there's a children's tumour foundation run out of a medical research institute in Melbourne, but I haven't called them. Our Paed Neuro is one of the best for NF, he's aligned with Harvard/Boston Children's Medical and I've full confidence in him- but I haven't seen him since August. The rest of the testing he ordered needs to be done before we see him again- and apparently then we get the full force of the NF clinic at Royal Children's, including parental support.

The waiting is hard. I have to take this in small bites and not look too far ahead. I can't bear the idea of multiple surgeries on his leg (although with the tibia discrepancy... that's on the cards). Or his body or face being covered in fibromas. He's such a beautiful little boy who's just learning his place in the world.

Thanks again, I truly appreciate your kind input.

OP posts:
remindmewhyidothis · 13/01/2022 06:42

Hi my DD has it (age 12). It was a complete surprise to us, we don't know anyone else in our family who has it. As everyone has said, it's really, really varied in how it presents. DD has no learning difficulties and is quite academic and creative. She was very slow to learn to swim and ride a bike and is now displaying anxiety and extreme emotions (which could equally be puberty / covid related). She has all the markers of NF1 but little or no symptoms up till now. She has no visible fibromas at present. However she's now under investigation for recurrent episodes this year of blurred / double vision which I'll freely admit is quite a worry. She was first diagnosed aged 2 and in terms of dealing with it all I could do was park the future worries and deal with what was in the present. I wasn't sure from your last post, are you in the UK or US? (I'm wondering how differently NF1 is monitored in children if so?)

remindmewhyidothis · 13/01/2022 06:44

Sorry OP, I just realised your first post said you're in Australia

Angel2702 · 13/01/2022 06:49

My daughter is on a very long watch and wait since age 3. She is now 10. She has numerous large cafe au lait marks but as they are all confined to one side of her body they suspect it is mosaic NF1 so doesn’t show up in the genetic testing. We were offered a biopsy of one of the cafe au lait but decided against it since it won’t change the outcome.

30not13 · 13/01/2022 06:51

I used to look after a child 13 years ago who
went on to be diagnosed with this although I can't recall if it's 1 or 2.

I know the family get support from Funny Lumps

Flowers
Kaiken · 21/01/2022 09:00

@Dustyblue rare diseases' support groups are usually run by patients themselves because rare diseases are neglected by the medical establishment and Nf1 is no exception.
Patients with rare diseases have no choice but to become expert in their disease because most doctors will know nothing about it or the best way to care for someone with that disease.

Nf1 specialists push nf1 patients to educate themselves and advocate for themselves. So don't be too quick in dismissing the patient-led online groups since they are probably the best source of recent information.

These online groups will be able to teach you how to download medical books for free or how to bypass the paywall on medical papers on pubmet. So give them a chance before judging them. I am not talking here about "Facebook" groups or similar which are trash , but more groups like Inspire.

Australia' s Nf1 foundations are CTF and flicker of hope in Melbourne, both heavily centred on fundraising and very thin on science. You are better off using the American CTF site www.ctf.org or the NF Network www.nfnetwork.org , both have webinars that cover several topics. They also organise masterclasses and medical symposium. There is one next week on paediatric management ctfeurope.org/research/masterclasses-in-nf you can enrol for free. Just type " none" on the enrolment form when it asks for affiliation, institution and stuff like that. A couple of webinar from NF Network you should watch from their library www.nfnetwork.org/resources/webinars/ is :

  • this one From the director of the biggest NF1 clinic in the US
and this one even if it is older

Australia is quite behind in the management and care of nf1, especially on the prevention front. It is still vey much reactive, which is a pity given that they are steps and drugs one can take to minimise the severity of the manifestations.

This brings me to your comment on diet and it shows that you don't have an understanding yet about the disease. The Nf1 gene produces a protein called neurofibromin which regulates the life cycle of the cell.

When not enough neurofibromin is circulating because the gene is mutated, several processes of the life cycle of the cell are malfunctioning, be it cell proliferation, migration, and even programmed death. Neurofibromin also impacts the metabolism and people with nf1 have an abnormal lipid metabolism , an abnormal glucose metabolism, higher inflammation and higher oxidation.
A diet that take these characteristics into consideration will help the cellular pathways affected.
It has been known for more than a decade that inflammation is needed for tumour initiation. An anti-inflammatory diet is therefor recommended.

There is a drug that can reduce the amount of neurofibromas one will get. It is called Ketotifen and it is a very safe , very cheap, very old allergy drug that regulates mast cells. Neurofibromas are mainly made of mast cells. Ketotifen in the pill format is not available in Australia as a normal prescription but can be ordered from Japan for less than $15 or made in Australia in a compounding pharmacy at roughly $100

What you describe for your son's leg is called long bone dysplasia and is far from rare in nf1. Again it has to do with the protein neurofibromin that lays a significant role in bone homeostasis and bone development (through a signal transduction pathway called Ras/MAPK) .

They will be many challenges and you will often be faced with medical apathy, treating Nf1 the way it was treated in the 1980s.

Nf1's manifestations are age-related, meaning they have a specific age of onset. At your son's age, the biggest one is the optic pathway glioma (OPG)

Has your son had an head MRI to check for the absence/presence of optic glioma ? Not all glioma are symptomatic and depending on the location, it can be missed with an eye observation. The role of systematic MRI screening for OPGs in children with NF1 has been widely discussed in the literature and is controversial and most countries still apply 1997 guidelines which rule against . However recent studies have shown that chiasmatic and postchiasmatic OPGs carried the highest risk for progression and vision loss and that early identification with MRI screening in asymptomatic cases may lead to improved visual outcomes.
If you are not familiar with these term, the chiasm is the part of the brain where the optic nerves cross. A child with Nf1 can develop an OPG on the optic nerve before the chiasm or after. Those after can be missed in an eye examination.

A good balance would be to have 1 MRI to rule out post-chiasmatic OPG

Nf1 can be managed . The gene mutation itself is not enough to explain what causes problems and this is why two individuals with the exact same mutation ,will have dramatically different outcome. The Pearson twins (vimeo.com/315774991) , with identical DNA and identical NF1 mutation are a good example. So if it isn't in the gene alone, what are these gene modifiers and can they be influenced? The immune system plays a big role, as does some of the other metabolic features I was mentioning before.

I would suggest you spend some time on the US sites I linked for education and use CTF Australia for this support in navigating the Australian system.

You need to know that for most issues, there are options.
As a example, let me cite hypotonia and wetness, clumsiness. Because of the abnormal lipid metabolism, dietary fats are stored in muscles and not fat cells, resulting in poor muscle tone but taking L-Carnitine as shown by the studies of Aaron Schinder in Australia, will rescue this myopathy. Ideally however , since people with NF1 do not metabolise fat properly, it would be recommended to avoid greasy, fatty and fried food. Hence the diet aspect being important on many fronts.
Just an example amongst many to show that there is more to the NF1 story than you suspect.

TonywithNF2 · 16/07/2022 11:04

I'm in south Australia & have NF2 with 40 years experience in tutoring senior medical students about this condition. Can I be of assistance?

Kaiken · 16/07/2022 12:48

That's very cool Tony , how do you do that? Do you only do it for NF2?
Nf1 is a very different disease.

Dustyblue · 16/07/2022 12:55

Hi All

Amazed to see this thread revived! DS6 most certainly has NF1. We're lucky enough to have seen some fabulous specialists at the Royal Children's Hospital in Melbourne.

Eyes, bones, development, genetics, it's a freaking whirlwind!

Yes, NF1 is very different to NF2.

Sorry to be brief, it's almost bedtime here.

OP posts:
Kaiken · 16/07/2022 12:58

Hej OP!

come back tomorrow and tells us more!

Dustyblue · 17/07/2022 04:19

@TonywithNF2 Thanks for your message. We're in Sth Gippsland, Vic. When DS6 was first suspected to have NF at age 4, it was assumed it'd be NF1. I'm unsure why, perhaps early symptoms of NF2 differ, or just because NF2 is less common?

@Kaiken thanks for your note, here goes...

It turns out we started the diagnostic process backwards, in a sense. When it was first suspected I arranged genetic testing, which came back with a detected mutation associated with NF1. Next (through family contacts) we saw Gabriel Dabscheck, Paed Neurologist who runs the NF clinic at RCH. He said kiddo almost certainly has NF1.

Then we proceeded to testing- opthamologist to check for optic gliomas and lisch nodules (no gliomas, one nodule). Orthopaedics to examine his legs (one longer than the other by 20mm), then genetics for family history etc. Turns out we'd have been better off starting with Genetics- by that time I already knew what I was dealing with and became quite frustrated with the Genetics people. I had to apologise when I left!

He started school this year and has had many issues. He can't use his hands properly (dysgraphia) and still hasn't chosen left over right. Behavioural stuff is coming to the fore also.

Next up is a new Paediatrician (they are rare in these parts!), a speech therapist (I hadn't noticed issues with his speech, but it's recommended) and a Paediatric Dietician. Luckily there's a "Fussy Eating Clinic" only an hour away. And continuing Occupational Therapy.

Tony, you will know what I mean when I say I've secured NDIS funding (government disability insurance available in Australia).

I'd also be very interested to hear about your work if you can be bothered writing about it here?

OP posts: