Spinal Muscular Atrophy – Symptoms, Diagnosis and Patient Care

Seven-month-old Maja suffers from an incurable, fatal disease. In a well-developing child, the first symptoms appeared after standard immunization. Today, the only chance to improve the girl’s health is expensive therapy abroad.

– Maja is now seven months old. When was the first time you noticed something was wrong with your baby?

-It was in November – says Ewelina, Maja’s mother. – Maja was then given the first vaccine. She became very weak after her and stopped moving her legs, but we thought it was caused by the vaccination itself and she would be back soon. Unfortunately, it went on and on.

– Could it be the vaccine that caused the early symptoms?

– Yes, it is quite common in children with this disease – says Łukasz, Maja’s father – because after the vaccination the body behaves like after an infection, and it is during the infection that the first symptoms usually appear.

– The muscles just get weaker while fighting the virus. The fact that at first we were full of hope that it was nothing serious is due to the fact that I had a very difficult birth – says Ewelina. – Maja was born hypoxic and right after she was born she was all blue. So the doctors thought that her weakening might be due to some kind of cerebral palsy.

– Maja fell ill in December and we went to the doctor – continues Łukasz. “It was a typical cold checkup, but we said we were concerned that our daughter was starting to weaken. After the basic neurological examination, the primary care physician stated that it would indeed be good for her daughter to be examined by a specialist. At the time, however, we did not suspect at all that it could be such a serious disease.

– Did you get to the neurologist right away?

– We were given the date of visit for September, so we decided to examine our daughter privately, but the visit could not take place until January – says Ewelina.

– When we finally got to the examination, the neurologist got very scared. We were immediately referred to the hospital and went to the Children’s Memorial Health Institute. There it was immediately suspected that it was something serious and genetic tests were ordered. It remained to wait for the results. In fact, the results were in the hospital after a week, but they were lost somewhere, so everything dragged on.

– The wife found out that there was any suspicion of such a disease by accident – says Łukasz. – In the Silesian Children’s Memorial Health Institute, which is a university clinic, the doctor had a meeting with students. Ewelina heard through the closed door that it was probably a child with spinal muscular atrophy.

– Did it mean anything to you? Did you know what this disease is?

– We had no idea what the disease was and what it was related to. We continued to reject the thought that it could be something serious – says Łukasz. – We were very scared, but we did not suspect the worst. It was only after I searched the internet for what the disease was that I returned to my wife’s hospital the next day in a completely different mood.

– So you obtained the first information about what could be happening with your child by accident, and you found the rest yourself.

– In our case it was very difficult – says Maja’s father. – We waited a long time for this lost result. At one point, we called not even the doctor, but the hospital office, and thus we got confirmation of the worst – spinal muscular atrophy – just on a piece of paper. We had a huge grudge because we were able to start working much earlier.

– Later we met the doctor in the hospital corridor and she confirmed that it was the disease – says Maja’s mother. – My husband kept asking if they were wrong. It does happen.

– Later, we made an appointment with the attending doctor, who explained exactly what it involves, where we should go for help. Anyway – we were left alone with it – says Łukasz.

– That is, about three months elapsed from the onset of the first symptoms to the diagnosis. It is quite a long time.

– Quite a long time indeed, but we were lucky anyway. It is very rare for GPs to consider such a disease at all. After talking to the parents of other children suffering from SMA, it turned out that in our case it was not too bad, says Łukasz.

– Additionally, Maja is not in the worst situation. There are children who become much more weaker and at their age have respiratory problems and end up in the ICU. Then they are diagnosed very quickly, but they are simply in worse condition. Maja suffered only from muscle weakness, which is probably why this diagnosis lasted a bit longer for us.

– What did the doctor tell you when you finally met him? What will happen to your baby in turn?

– Maja has type I spinal muscular atrophy (SMA I), i.e. Werdnig-Hoffmann disease – explains the girl’s mother – it is the most severe type of spinal muscular atrophy. Children with this form of the disease live for about two years. The skeletal muscles atrophy first. Maja is already limp, does not move her legs or raise her head. The hands are getting weaker and weaker. Then, as a result of the weakening of the respiratory muscles, problems with lung ventilation arise. We are already connecting Maja to a respirator for 15 minutes a day so that she can get used to it. Swallowing problems eventually arise, so tube feeding is essential.

– Did you hear the answer to the most difficult question?

– The doctor did not tell us exactly what the prognosis was – says Łukasz. – She did not want to tell us exactly how much such a child can live, because each case is different. Unfortunately, such a conversation usually comes down to the question of how much time we have. In Poland, it is 2-3 years. However, today, when medicine is more developed, you can get a lot of the necessary equipment on your own or with the help of a foundation. The length and quality of life of these children is increasing.

– How has your life changed after the diagnosis?

– Before we got the final confirmation, it was clear that Maja had problems with muscle tone. We already had recommendations to rehabilitate her back then and we did it very intensively – says Łukasz.

– This time was an emotional swing for us. One day we cried because we knew what could happen, and the next day, when we saw how good Maja reacted, we completely rejected it. It was a hurricane of emotions. We were probably looking for signs that this is not the disease, we assumed a much better scenario.

– And when was the final diagnosis?

– We went through the worst two weeks. We took care of ourselves, our emotions. My wife and I cried in turns, we didn’t know what to do and Maja suffered from it. It was a lost two weeks because we had no motivation.

– Then we came to the conclusion that this is not the end of the world and no matter how long Maja’s life is, she should be happy. I think we recovered quickly anyway. As of today, we are doing everything to make her happy and to help her.

– What about your earlier life, with your friends?

– We had to limit the exit from the house – says Ewelina.

– Yes, we cannot be in public places at all, because it is associated with too high risk of infection, which may end tragically for Maja – adds her husband. – We have to avoid germs like fire. All our friends know that if they have any suspicion of the slightest infection, they are not allowed to enter our home. Sometimes there are times when we both have to leave, then usually one of us waits outside with Maya and we change. If you absolutely have to go somewhere with her, we just clean the area.

– We cannot leave anyone with her, because there is a risk that the breathing may stop at any moment – says Maja’s mother. – In this case, we have an ambu bag, a resuscitation device, and our daughter must be in the presence of someone who can handle it.

– We have undergone appropriate training and already know what to do, but acquiring this knowledge was not easy – adds Łukasz. – At first, we were so shocked by the fact that we even need to know it, that it was difficult for us to focus and absorb this information.

– Are there other things in everyday life that seem important, apart from Maja’s disease?

– Our approach to life has changed completely – says Łukasz. – We underestimate all other troubles that we encounter. What happened caused a huge change in our understanding of the world and what really matters.

– As a result, we also have slightly fewer friends – their problems seem just a bit funny to us. On the other hand, we have no right to prevent them from worrying about their own affairs. Their problems are as important to them as ours are to us. It’s just really difficult for us to meet someone for whom the end of the world is a cold or a cold. We feel awkward, but we also don’t want to embarrass others.

– Which is the worst thing about such conversations.

– Questions, what’s up, what’s the situation? Ewelina replies. – Because what can be answered to such a question?

– It cannot be summed up as “good” or “bad”. That’s why I have been saying for some time, if you want to know what’s going on with Maja, check out our website – adds the girl’s dad.

– We have heard comments many times, it will be good – says Ewelina. Łukasz explains: – Many people have no idea what this disease is and we cannot expect it from them. However, it is impossible to calmly listen to someone saying that it will be okay when they do not have any knowledge about it. It’s just awkward. We don’t expect too much from people, but it’s just too difficult for us.

– Maja spends most of her time at home. What is her day like?

– Our apartment has turned into a hospital room – says Ewelina. “We have a respirator, an oxygen concentrator, and a lot of other equipment that we have to use all the time.

– When Maja wakes up, we start with cleaning the upper respiratory tract – says Łukasz. – For now, ordinary sea water and a mammal are enough – this is the basic instrument for this disease.

– Then we practice – Ewelina continues. – We have to do this four times a day. Rehabilitation with a specialist is a drop in the ocean, we do a lot ourselves. After exercise, we usually rest, eat and play for a while. Later, a physiotherapist comes and does exercises as well. In the afternoon, we connect Maja to a respirator for 15 minutes so that she can get used to it.

– We constantly monitor the basic life functions pulse and saturation with a pulse oximeter. This is essential for every child with SMA.

– How does Maja react to all this?

– In fact, for Maja, even sitting on her knees is tiring, because there is no support on all sides – says Łukasz, taking her daughter on her lap. – Sometimes she is tired after half an hour just because her grandmothers come and take turns carrying her in their arms.

– You have to bear in mind that Maja is too weak to take care of herself, play herself. So it is enough to leave her for a while and she cries right away. You have to show her toys, wave to her.

– Maja has special toys, doesn’t she?

– Yes, her hands are too weak for her to play with ordinary stuffed animals, and like every child she wants to touch everything, put it in her mouth. That is why we try to get very light toys so that she can take them in her hands and feel like a normal child.

– The disease is incurable, but recently it has become possible to take part in therapy abroad. How can Maja help?

Medicines that can improve the condition of children with SMA have been intensively researched for many years. One of the drugs will enter the market in a year. This measure stops the disease from progressing, but it does not reverse the changes that have taken place in the body. Thanks to this, sick children may be looking forward to therapy that will help them in the future. Unfortunately, as I mentioned, the measure will be available in a year, and that is a huge amount of time for Maja.

– In Poland, we have already done everything we could. We regularly rehabilitate Maja, we are visited by doctors and a nurse – adds Ewelina. – However, no treatment is available in our country. We help her as best we can, but we have no hope of saving her.

– However, the prospect of treatment abroad has emerged that may halt the progression of the disease now, so there is hope that in the future it will not have to be permanently connected to a ventilator and fed with a probe. We already know that the measure gave very good results in the first tests – continues Ewelina.

“We asked our anesthesiologist what he thought about this treatment,” says Maja’s father. He replied that he knew about the drug and the research it involved. He told us one sentence. If I were you, I’d do the same.

– So the search for new methods of treatment was your initiative, not the doctors?

– Yes, it was entirely our initiative. One and a half months after the diagnosis, we had a checkup and the doctor told us that he was surprised by the amount of things we had done for Maja in such a short time and that she had not encountered anything like that yet. Although I consider the first two weeks to be wasted, we must have cried all the tears then and now there is only determination left – Łukasz continues. – Today we have a great understanding of what can be done for a child with SMA, and we are happy to provide information to all parents who find out that their child also has the disease.

– Will the National Health Fund support you in financing this therapy in any way?

– Unfortunately not – admits Ewelina. All costs related to travel, living abroad and organizing professional physical rehabilitation, respiratory and health care, and equipment remain on our side, and this is not a small amount of money. We hope that thanks to people of good will, we will be able to finance the trip, which is a great opportunity for our daughter.

If you want to help Maja, visit her website. There you will find the bank account of the foundation that the girl takes care of. http://majaadamczyk.pl

Tekst: Aleksandra Gołąb

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