My Son's Story

[Candace P]

I'm writing to tell about my 11 year old son, Jacob. He was diagnosed with sever AA this past June and had ATG in early July. He's homebound from school as his ANC is low. Luckily, he's a homebody and hasn't been too bummed about being on "house arrest". His last counts were hemoglobin- 7.8 (we're planning to transfuse this week), platelets 41,000 and WBC- 3.6 with an ANC of 700. Of course, last week the ANC was 250 so it goes up and down A LOT. He takes 100 mg of cyclosporine 2x per day. No siblings so no match and the doctor hasn't even hinted at a BMT and when I asked about another round of ATG he said " the body will determine that". My son goes to the largest children's hospital in Indiana and I know the doctors are good but I feel like his main doctor thinks I'm an idiot. I thought this site might be a safer place for me to ask questions. My son has daily migrating joint pain. Has anyone experienced joint pain from ANC? He could just be having a growth spurt. His appetite has begun to go down. Is this normal? the doctor suggested giving him a vitamin with iron but everything I've read discourages giving iron. I'd love to know of any other success stories regarding AA. Honestly, I'm needing positive reinforcements and to know that this disease can be beaten. It's nice to know we aren't alone!!

[missmel]

So sorry to hear about your son! My 17 year old was diagnosed with AA in Sept 2010. Received ATG in oct 2010 and started cyclosporine at the same time. Continues to take cyclosporine 300-350 twice daily depending on his cyclosporine level and kidney function. His counts have been improving every week without transfusions. Last platelet transfusion Oct 15 and blood Oct 29. We live in southern Illinois and go to St Louis Children's Hospital. The drs there are GREAT!! His doctor is very informative and answers all questions. Do you know how your child aquired AA? I always wonder how this has happened to my son, he has always been a perfectly healthy kid. My other question about your child is are any of their counts rising? I will keep you and your family in my prayers.

[missmel]

I forgot to answer a few of your questions. My son had severe pain in his knees for about a week. Dr said he could take some tylenol and this seemed to help. No more complaints but sometimes I think he doesn't think about his illiness and goes on with life like a typical teenager. I do believe that one of the side effects of either cyclosporine or ATG is joint pain. Have you talked to the dr about this. This is great for him as I want him to have a normal life especially at such an important time in his life senior year of highschool. I'm the worry wart constantly questioning him. His appetite has decreased dramatically! He used to eat so much I could hardly afford to feed him. We never had leftovers. Now he eats much less, everytime I ask him if he's hungary he says not really. Hope this helps.

[evansmom]

Hi there,

Sorry to hear that your son is having to deal with this awful disease. My son was diagnosed with AA in November 2007 at the age of 10. He was a non-responder to ATG and went on to have a successful unrelated BMT in June 2008. We were very lucky as we went from diagnosis to full recovery in 13-14 months. Evan's complete journal start to finish is linked below. Feel free to visit. This disease is successfully conquered all of the time, keep the faith!

My son would get bone/joint pain in the months after his ATG treatment, but I first attributed that to serum sickness, despite being on prednisone. When it continued, I attributed it to marrow stimulation, especially because he was on G-CSF injections to artificially raise his ANC.

You are right not to give your son any extra iron at this time. He'll be getting more than his body can use through the red cell transfusions.

You have found extended family here. This forum was one of my life lines during our time with AA.


Best wishes for complete return of health to your boy,

[Neil Cuadra]

Candace,

Jacob's doctor doesn't sound like much of a communicator. Have you considered getting a second opinion from another doctor? You can bring previous test results so Jacob won't have to endure more tests, and hearing what another doctor thinks will give you a better picture of the reasonable choices of treatment (one choice being "wait and watch"). A good pediatric hematologist ought to be willing and able to answer as many questions as you want to ask.

[Candace P]

Originally Posted by missmel

So sorry to hear about your son! My 17 year old was diagnosed with AA in Sept 2010. Received ATG in oct 2010 and started cyclosporine at the same time. Continues to take cyclosporine 300-350 twice daily depending on his cyclosporine level and kidney function. His counts have been improving every week without transfusions. Last platelet transfusion Oct 15 and blood Oct 29. We live in southern Illinois and go to St Louis Children's Hospital. The drs there are GREAT!! His doctor is very informative and answers all questions. Do you know how your child aquired AA? I always wonder how this has happened to my son, he has always been a perfectly healthy kid. My other question about your child is are any of their counts rising? I will keep you and your family in my prayers.

It sounds like we've have a similar year. It sounds like your son is doing awesome. ATG and then no transfusions within weeks is amazing. Jacob's counts have gone up SLOWLY. His His platelets topped 40,000 last week and he's scheduled to get a red transfusion this Thursday, but the last one was 6 weeks ago. He tolerates 100 mg of cyclosporine 2x per day and I often wonder if that's too low of a dose to make a big difference. But we have to watch his kidneys so if that's all he can tolerate we can't do much else. I'm so glad to have found this site! My husband worked near Muscoutah last year and we almost moved to southern Illinois but he found a job close to home. Thanks for responding. Oh yeah, I have no idea how Jacob got AA. We did quit using grass fertilizers this year since we're on a well. They told us we'll never know what caused it which is frustrating. Is your son going to school? We have a teacher come to the house 2x a week. Jacob can go back once is ANC is over 1000.... hopefully in a month or two.

[Candace P]

Originally Posted by evansmom

Hi there,

Sorry to hear that your son is having to deal with this awful disease. My son was diagnosed with AA in November 2007 at the age of 10. He was a non-responder to ATG and went on to have a successful unrelated BMT in June 2008. We were very lucky as we went from diagnosis to full recovery in 13-14 months. Evan's complete journal start to finish is linked below. Feel free to visit. This disease is successfully conquered all of the time, keep the faith!

My son would get bone/joint pain in the months after his ATG treatment, but I first attributed that to serum sickness, despite being on prednisone. When it continued, I attributed it to marrow stimulation, especially because he was on G-CSF injections to artificially raise his ANC.

You are right not to give your son any extra iron at this time. He'll be getting more than his body can use through the red cell transfusions.

You have found extended family here. This forum was one of my life lines during our time with AA.


Best wishes for complete return of health to your boy,

You've definitely injected me with some hope! I don't want Jacob to have a BMT if it can be avoided but it's nice to know it can be successful with a non-relative donor. When you say your son didn't respond to ATG, what does that mean? Did his counts go up any? Was he still needing weekly transfusion? Why didn't they do it a second time? What hospital did you go to? If Jacob can end up healthy like your son Evan I will be ecstatic! I just have to hang in there a little while longer. Jacob has weathered this illness well. He rarely complains and he doesn't mind being stuck at home. I have serious "cabin fever" but know it will all be worth it once he's back to his old "normal". Thanks a lot!!

[Candace P]

Originally Posted by Neil Cuadra

Candace,

Jacob's doctor doesn't sound like much of a communicator. Have you considered getting a second opinion from another doctor? You can bring previous test results so Jacob won't have to endure more tests, and hearing what another doctor thinks will give you a better picture of the reasonable choices of treatment (one choice being "wait and watch"). A good pediatric hematologist ought to be willing and able to answer as many questions as you want to ask.

I've considered switching to a different doctor within the hematology department. We currently see the director so I know he's a smart guy, but he's almost condescending to me. And I want to like him since he's handsome (ha ha) but he's making it difficult. When we first got the diagnosis of SAA I started to cry. Not a loud boo hoo but I did say "no" when the doctor said the words. He said "you can go, you're not helping". I knew then that I was not going to mesh with this guy. But, if he can make my son well again I'll be eternally grateful.... and maybe he'll be even more handsome then! Thanks for your response. This site is a life saver!!

[evansmom]

Evan had horse ATG, 5 doses over 5 days, followed by prednisone and cyclosporin. The prednisone was tapered out after a month or so and the cyclo, of course, continued up to transplant. In the month after diagnosis and before the ATG, Evan did not need transfusions. His hgb stayed around 80 and his platelets around 20. ANC was around 0.6. In the weeks that followed ATG, Evan was his most transfusion dependent. He needed platelets every 4 days or so and red cells every 2 weeks. Around 2 months post ATG, Evan settled out again and stayed low but stable without transfusions. He went back to school with hgb 85, platelets 25, ANC 1.2. Then in March 2008, 4 months post ATG, his counts plummeted: hgb 64, platelets 8, ANC 0.3. From that point, he needed very frequent transfusions. He was back out of school and we were talking transplant. On a side note, looking back, I believe the reason for his counts tanking was because cyclosporin was causing damage to his tiny blood vessels, causing his red cells to break apart, not because of advancing or worsening AA. I believe this because post BMT, Evan had a similar reaction to tacrolimus, a sister to cyclosporin in the class of calcineurin inhibitors, which caused a very serious drop in hgb i.e. from 84 to 48 in 3 days. While the clinic thought this was OK, I knew it was not. Pushing for investigations, we came to realize this rare and serious side effect (microangiopathy). Once we switched him to a different class of immunosuppressant (MMF), he recovered rather quickly and never looked back from there. Word of advice as far as this point is concerned: if at any point your son's hgb starts dropping uncharacteristically, suspect microangiopathy and ask questions. Diagnosis is confirmed by red cell fragments or schistocytes on the smear, low haptoglobin and very high LDH. The urine will be tea or rusty coloured. I tell you this because it is rare and rare = many doctors not recognizing it until damage is done or patient's condition becomes serious.
I sometimes wonder if we had recognized this condition happening before BMT and switched meds, that maybe Evan would have been a responder to ATG. I'll never know, I'm just so thankful that it all worked out alright.

We were given the option of a second round of ATG (rabbit) or doing the BMT provided the potential donors were still available. By this point, I had already read studies that indicated that AA kids have better outcomes if after 6 months of no response to ATG, BMT is done (again provided suitable donor available) versus doing ATG again. For us, it was an easy choice because we never saw his counts climb. There was no response. So in our minds we thought if one round of ATG didn't work, why would we believe a second round would work? Maybe it would...but maybe it wouldn't. We had a donor ready and willing and we didn't want to waste precious time trying something that may not work, especially knowing more immunosuppression therapy and more transfusions = more potential complications in BMT.

Prospective multicenter trial comparing repeated immunosuppressive therapy with stem-cell transplantation from an alternative donor as second-line treatment for children with severe and very severe aplastic anemia.

Kosaka, Yoshiyuki. Yagasaki, Hiroshi. Sano, Kimihiko. Kobayashi, Ryoji. Ayukawa, Hiroshi. Kaneko, Takashi. Yabe, Hiromasa. Tsuchida, Masahiro. Mugishima, Hideo. Ohara, Akira. Morimoto, Akira. Otsuka, Yo****oshi. Ohga, Shouichi. Bessho, Fumio. Nakahata, Tatsutoshi. Tsukimoto, Ichiro. Kojima, Seiji. Japan Childhood Aplastic Anemia Study Group.


Blood. 111(3):1054-9, 2008 Feb 1


We conducted a prospective multicenter study to compare the efficacy of repeated immunosuppressive therapy (IST) with stem-cell transplantation (SCT) from an alternative donor in children with acquired aplastic anemia (AA) who failed to respond to an initial course of IST.

Patients with severe (n = 86) and very severe disease (n = 119) received initial IST consisting of antithymocyte globulin (ATG) and cyclosporine. Sixty patients failed to respond to IST after 6 months from the initial IST and were eligible for second-line treatment.

Among them, 21 patients lacking suitable donors received a second course of IST. Three patients developed an anaphylactoid reaction to ATG and could not complete the second IST.

A trilineage response was seen in only 2 of 18 (11%) evaluable patients after 6 months.

Thirty-one patients received SCT from an alternative donor.

At 5 years from the initiation of second-line therapy, the estimated failure-free survival (FFS), defined as survival with response, was 83.9% (+/- 16.1%, SD) in the SCT group compared with 9.5% (+/- 9.0%) in the IST group (P = .001).

These results suggest that SCT from an alternative donor offers a better chance of FFS than a second IST in patients not responding to an initial IST.

Arm yourself with as much information as possible, you have involuntarily enrolled in a Bone Marrow 101 rapid study course.

[Greg H]

Originally Posted by Candace P

When we first got the diagnosis of SAA I started to cry. Not a loud boo hoo but I did say "no" when the doctor said the words. He said "you can go, you're not helping". I knew then that I was not going to mesh with this guy.

Candace,

I'm surprised you didn't deck the guy right there; you have way more will power and self-control than I could ever muster. What a jerk this guy is!

I understand that you may have decided to put up with him because you feel that's best for your son. That's your decision and you have every right to make that decision.

But you also have every right to fire this guy and find a doctor who knows how to treat patients and caregivers with respect.

He's your son, your his Mom, it's your decision. You're the boss. Don't ever let anyone tell you any different. Even if he is smart and good looking.

Take care!

Greg

[Lisa V]

Candace, I'm with Greg on this. There are so many things you have to battle with in a rare disease, your medical provider shouldn't be one of them. If he treats you condescendingly or doesn't give you the information you ask for, file a complaint. He may be the director of the department, but I'm sure he also has a supervisor who evaluates his performance. There's more to being a good doctor than just medical expertise.

My husband's hem/onc retired recently and we got a replacement who had the bedside manner of a pile of rocks. He may have known his stuff, but he got bad reviews from his patients so he's out. HIS replacement is an absolute doll. One meeting and it was obvious we're all on the same page. Color me happy!

BTW, I'd also like to give a big thumbs up to Nicole for giving such good advice (as usual)!

[evansmom]

Thank you Lisa. Your knowledge and insightfulness is very appreciated as well.

[Candace P]

Nicole, what an amazing study you forwarded to me. Those statistics were a surprise and when seeing them it's a no-brainer to go with the SCT. Was it a long wait to find a donor? We haven't started that process yet. I'm really considering switching to a different doctor. I need to remember the staff in essence works for us and we deserve to have every little question answered. I'm going to go into to Thursday's appointment with a new attitude. Thanks for the information, encouragement and support! Candace

[evansmom]

Hi Candace,

Hopefully your son won't need to go to transplant but knowledge is power. Once we decided to pursue transplant, the BMT doc already knew of 3 potential donors - 2 cord blood units and one adult. We were told about the various mismatches of each and were told to go home and think about which source we would like to secure. Because the mismatch with the adult was less serious than that of the cords, we went with the adult. From that point, we were admitted for BMT in 8 weeks. It does very much depend on how long it takes to contact the donor, how quickly he or she can drop things and get their labs and physical done and whether or not they are healthy enough to donate.

I encourage you to speak your mind to your son's doctor and let him know what your expectations are. Your son is 5 months out from ATG now and it would be prudent to have the next steps planned out. A donor search should be done now, in my opinion. My greatest concern is his ongoing dips in ANC below 500. Your son cannot afford to get a fungal infection and the longer he sits below 500, the higher the risk.

Dr. David Margolis at Children's Hospital of Wisconsin has fixed many kids with AA through BMT and ATG. He's the pediatric expert and if you send him an email and include your phone number, he would likely get back to you ASAP. I consulted with him many times, even though we are up here in Canada. I don't know how easy it would be for you to temporarily relocate to Milwaukee, but I can tell you he'd likely see your son and secure a donor (if he felt it was the treatment of choice) in no time flat. I've met up with him several times at AA conferences and he is just wonderful.

Time is ticking, you son still needs red cell transfusions and his ANC is in the risky zone. At this point, you need to know what's next.

Dr. Margolis:

http://www.chw.org/Applications/PPF/...ysHomePage.asp

[Candace P]

Originally Posted by evansmom

Hi Candace,

Hopefully your son won't need to go to transplant but knowledge is power. Once we decided to pursue transplant, the BMT doc already knew of 3 potential donors - 2 cord blood units and one adult. We were told about the various mismatches of each and were told to go home and think about which source we would like to secure. Because the mismatch with the adult was less serious than that of the cords, we went with the adult. From that point, we were admitted for BMT in 8 weeks. It does very much depend on how long it takes to contact the donor, how quickly he or she can drop things and get their labs and physical done and whether or not they are healthy enough to donate.

I encourage you to speak your mind to your son's doctor and let him know what your expectations are. Your son is 5 months out from ATG now and it would be prudent to have the next steps planned out. A donor search should be done now, in my opinion. My greatest concern is his ongoing dips in ANC below 500. Your son cannot afford to get a fungal infection and the longer he sits below 500, the higher the risk.

Dr. David Margolis at Children's Hospital of Wisconsin has fixed many kids with AA through BMT and ATG. He's the pediatric expert and if you send him an email and include your phone number, he would likely get back to you ASAP. I consulted with him many times, even though we are up here in Canada. I don't know how easy it would be for you to temporarily relocate to Milwaukee, but I can tell you he'd likely see your son and secure a donor (if he felt it was the treatment of choice) in no time flat. I've met up with him several times at AA conferences and he is just wonderful.

Time is ticking, you son still needs red cell transfusions and his ANC is in the risky zone. At this point, you need to know what's next.

Dr. Margolis:

http://www.chw.org/Applications/PPF/...ysHomePage.asp

You're a wealth of information and I'm feeling extremely lucky to have found this site so I can gather much needed advice and information. We go to the doctor tomorrow so hopefully we'll establish a plan B,if needed. The doctor just keeps saying "It will take a long time" for the counts to come up. I'll keep you posted. THANKS !!!!!

[evansmom]

Just let the doc know you are no longer comfortable sitting back and waiting. If your son's ANC was always above 500 and he didn't need transfusions, then waiting would be appropriate but this is not the case. A fungal infection could settle into the lungs when the ANC is really low and then we'd have a really big problem.

Good luck.