AA

[trishgen2001]

My daughter, Jessica, age 16, was diagnosed about 2 weeks ago with Moderate AA. We're still in shock, but trying to research and read (and understand!) everything we can get a hold of. Right now, the treatment plan is 6 - 8 weeks of monitoring blood counts, then ATG & Cyclosporin. (she doesn't have a sibling for a matched bmt, or that would have been her Drs. first choice) The treatment sounds pretty scary, and we are so afraid of making the wrong choices. Any advice is appreciated.

[Jbsx19]

Trish,

Hi, I am so sorry about your daughter's diagnosis and certainly understand how scared and overwhelmed you are right now trying to learn and research everything there is to know about AA. My story is posted here already. I was diagnosed VSAA in 2003 when I was 41yoa, a wife and mother of two. I was treated at NIH by Dr Neal Young ( the pioneer in AA treatment and research). I would suggest that since you are so close to NIH to at least go there and be evaluated before making any treatment decisions. Once you get treatment elsewhere, it may limit your daughter's options for treatment at NIH or in a clinical trial of some sort. Whether insurance is or is not an issue for you, my treatment did not cost me a cent. All drugs were supplied to me at NIH and the GCsf(neupogin) was overnighted to my home. All my subsequent flights,hotels,etc are covered by NIH and AAMDS will help with initial costs of travel to NIH. You can research treatment options at www.NIH.gov and even see quite lengthy lecture by Dr. Young about AA and treatment. If you can't find the video, I have it and can email it to you.

Again, sorry you are in this situation,too...but know that you need to stay strong and focused. Knowledge is power and there are plenty of us out there to help you through that have been through it as well.

Hang in there....Judi

[trishgen2001]

Judi-Thank-you for your response. I do have contact info for nih and I will be calling them today. If there is anything good about this, it is our insurance. We don't have the worry of trying to pay for all of this. We just want what is best for her regardless. I always wondered how parents handled such news about their children's health. Now I understand you just DO what has to be done. Jessica is a trooper and has been very brave so far. We're staying strong and positive for her. How did you handle your treatment, ie the side effects, how long did it take? I just need to know what to expect. Her Dr. has been wonderful, taking all my phone calls, answering the same questions patiently, and has explained all of this. But again, she's never had AA or gone through the treatments herself. I think just reading and talking to others who have been there,done that will be a big help.
Trying to hang in there!!

[Jbsx19]

Hi Trish,

Glad to hear you are being strong and researching. Contact AAMDS also and have a package sent to you which contains 1st rate booklets to explain everything about the disease and treatment options. My case was very swift indeed, I was diagnosed May 9th,2003(healthy the 8th and 0 platelets and white cells ,Hemoglobin like 5 on the 9th in the ER)BMB that day.They kept me in ICU to support me until all the contacts and testing on my sister could be done. She is 1200 miles away ,but a complete match.(I have 1 sibling with a 25% chance of matching..crazy,huh?)Anyway, flew up Mon,had BMB Tues.They admitted me right then..I had to decide between BMT and ATG/CYcl clinical trial. They did not have a trial at that time that went right to transplant at NIH. I had the best Dr on each side of the fence as to what treatment to do.They don't like to transplant after 40. In order to stay at NIH, I opted for the ATG and then they would do the transplant there if the ATG didnt work.I started ATG on May24th (4 day infusion)..no really bad side effects. A little flu like,chills,etc. I had a nose bleed that wouldnt quit that was the worst. Had to go to the ENT there to cortorize it. You have to understand that at NIH, that's what these nurses and NP's do is bone marrow.I think they may know as much as the DR's there. Honestly, it's a different medical world than your local.I had a nurse,literally, by my side all 4 hours of the ATG for 4 days.The nursing is 1 on 1. You get your own nurse for a 12 hr shift.I was assigned a whole team to take care of me and answer all questions(a nutritionist,therapist,activities(like crafts),massage,etc..etc...)I developed some serum sickness that even baffled Dr. Young,my hands and feet peeled,my white cells started to come up within a few days, i still couldnt hold onto to platelets at all(had to get HLA matched platelets)and only need very little red cells. I had a central line in my neck the whole time I was there,once I decided to do the ATG.I could shower everyday,walk around,at NIH they prefer you to dress ,do laundry,go to the cafeteria(with a mask if needed), no laying around in your PJ's.I responded very quickly and by June 9th my ANC was 900 (enough to go home). They infused HLA platelets the morning of the 10th and my husband picked me up and we drove home to FL,with a suitcase of drugs. Yes, I stayed up there for a month by myself, I needed my husband home to work and take care of the kids and house. My platelets were 28K on my ride home (they called to tell me). I got home, was tired,weak.and alot of bone pain Cyclosporin and Mycophenolate are immunosuppresives (along the same idea of chemo but not)I did Cyclo for 6 mos and MMF for 18 mos.Not fun side effects, but not hateful.Neupogin shots every 2 or 3 days(my husband gave me) and Pantemedine treatments once a month(breathing in of the drug to protect lungs from fungal pneumonia)Blood work every 2-3 days for a while. Back to NIH at 3,6,9,12 and 18 months. I went into full remission and never needed blood products again one I got home.It's been 5 years and my platelets are at 302K last week.I did enter another trial after to see if they could harvest my own stem cells(CD34) aftr remission, but I cannot> I had 0 CD34 cells to harvest after a week of Neupogin shots. They can't explain it at NIH,either. Sometimes you just have to accept what is and not look for an explanation.

When you call NIH, talk to OLga at NHLBI..I will be going up for my BMB on May 6th.I have her direct number if you need it. I will be glad to help you in anyway I can.My point in all of this, is that if you are unfortunately lucky enough to be diagnosed with something that will get you into NIH, why wouldn't you take that opportunity and be treated by the place who does this., and help them gather information for research at the same time.This is what they do there...treat bone marrow disease...not fit you into some oncology unit or lump you with cancer patients. A whole different world, honest!!

I can also deeply feel your pain with this being your child..it was me that was sick..not my child..That's a whole different ballgame when you are a parent.My 27yoa niece is losing her battle with a brain tumor,as we speak...I really do understand.

Email me and I will gladly give you my number if you need to talk.

Be strong..and hope I helped in some small way.

Judi...Jbsx19@aol.com

[Jbsx19]

Hi again,
I'm so glad to hear that you have insurance and didn't really think of this in my last post.
One thing I did think of that you should consider when making your decisions for treatment is this: Your daughter is 16yoa and under your insurance,wonderful. But , and I'm trying to be realistic for you,..Once an Aplastic Patient..always an Aplastic patient.If you choose ATG, A patient can go into remission and stay that way forever, but..you still have AA and still need to be monitored for the rest of your life. If you go the transplant route, the AA may be cured..but you are still a transplant patient..for the rest of your life. Once a patient at NIH, whether ATG or transplant,..you are a patient for life and will be monitored and treated...for life.Your daughter's insurance situation may change during the course of her long and healthy life(God willing)..adulthood,job,marriage,whatever(then it becomes a "preexisting condition")...but she will always know that the AA will not be a financial issue.
Just something to consider.. I know because I just got turned down twice for new Insurance because I am not 10years out yet...they rate AA as if it were cancer....

Hope you are coping well,

Judi

[trishgen2001]

Hi Judi~
Very good point on the insurance. I guess I'm just still having a hard time accepting the fact that this isn't something that's just going to go away, no matter how much I wish. About clinical trials.......I'm not sure that I'm willing to put her through that. I mean, it's hard enough using what they know to be effective, how will I know that we're not going to put her through even more than she's facing anyway? I really haven't read much on the clinical trials, just for that reason. But, I will. I guess bottom line is we won't quit. On the other hand, until a couple of weeks ago, I didn't realize how thankful we should all be for the people who are brave enough.
I'm very sorry to hear about your niece. I have two nieces myself, and I love them like my own.
Another question for you...had you been feeling bad before your blood counts dropped so much, or did it happen that suddenly?

[Jbsx19]

Hi Trish,

Thanks for thinking of my niece at a time like this for you. Part of the reason I am here is to help clear up some of the misinformation regarding clinical trials. At NO time will your daughter's health be at risk in a clinical trial at NIH. These are not "placebo" type trials. Depending on the trial they decide to put her in based on their own re-diagnosis, She would be given the standard ATG/CYclo and then a clinical trial drug that they may be investigating to see if it effects the outcome(maybe quicker response,maybe longer remission, )..Poss chemo instead of ATG..poss right to transplant...But you have the Best of the best making that decision on how to treat her specifically. Dr Neal Young (ATG,AA etc..) and Dr. John Barrett or Dr Childs (transplant) There are teams of doctors and fellows making rounds every day..I had probably 10 drs everday and then Grand Rounds once a week..doctor on the floor 24 hrs a day..my 2nd nite there a Dr sat with me all night talking and helping me decide what to do.
Anyway,..I was completely healthy May 7th (best shape of my life)..May 8th felt tired,out of breath walking to the mailbox(I had been walking 5 miles 3Xweek) and "funny little purple dots on my arms and legs"(petechia)May 9th woke up and my head was "funny" could almost hear my heartbeat in my head(no red cells..no oxygen to the brain) and then went to the bathroom..yikes..I apparently was bleeding internally..no nosebleed,etc..called my Husband home from work and went to the ER. They were shocked that I walked in..didn't "look" sick..then they got my CBC results and ...they were freaked..I was freaked..and hung platelets and took me up to ICU..with no platelets you can bleed out,have a brainbleed,etc..and with no white cells, your own body can kill you..we all have fungus and bacteria that normally our white cells kill off..without them..well not good..It's called neutropenic..Had to take my temp every 2 hrs (at home that weekend before NIH) super antibiotics on hand in case I went over 100.2..take one and rush to the ER.. you have a window of like 2 hours..special diet (no yogurt,fruit,fish,fresh veg,anything that might have bacteria)..so that's my story..and 5 years later I am sitting here able to tell it..What a gift, after being told even at NIH that they weren't sure I was going to make it.
So , I know the whole " Clinical trial" thing sounds scary...not to worry..honest..and NIH..WOW..what a place and since I was treated there they have built a whole new hospital..unbelievable..all private room, hardwood floors,flat screens/computer,no hospital food...you call the cafeteria when you are hungry and they bring it to you, I could go on and on..I mentioned the nursing care already..That's what these nurses do..bone marrow patients..that's it..NIH is your government at work for you...There is also shuttle service from the hotel to NIH or the airports...

I am here to help if you need anything......Judi