Issue: # 44
November 2014 
In This Issue

20 Years and Going Strong! 

It is hard to believe that we are closing in on the end of our 20th year. With the sweat and tears, dedication and hard work, sacrifices, commitment, and generosity of our families, friends and supporters, the Diamond Blackfan Anemia Foundation has accomplished many things over two decades. 

This past year, we have adopted our new Mission Statement:

 The mission of the Diamond Blackfan Anemia Foundation is to advance research initiatives that promote a better understanding, therapeutic strategies and a cure for this rare bone marrow failure syndrome. We are dedicated to providing patient advocacy, support and education services to individuals, families and medical professionals resulting in improvements in the diagnosis, clinical treatment and management of the disorder, while enhancing the quality of life of affected families worldwide.

We have funded major research projects, sponsored and co-sponsored numerous scientific meetings, sponsored international family retreats, attended ASH conferences and provided educational materials to medical professionals.

Above all, our goal is to serve our families. We are grateful for your involvement and appreciate the opportunity to connect with you. You are encouraged to reach out to us. We would love to hear from you!
Dawn Baumgardner


Upcoming Events

Friends of DBA 

Night at the Races
February 20, 2015
Weymouth Country Club
Medina, OH
Jim & Carol Mancuso

In Honor of Audrey
March 14, 2015
St. Edward Parish and School
Jeffersontown, KY
More information: 
Coming Soon 

Ongoing Fundraisers
Family Letter Writing Campaign  
Pre-printed letters and envelopes have been created for you to send to your contacts! Call or email for more information.
Dawn Baumgardner


Wristbands Available 
Twila Edwards




Tribute Cards Available
(3 Styles)
In honor of...
In memory of...
Holiday giving...
Dawn Baumgardner 
  donation donation
5" x 5" Stickers Available
Dawn Baumgardner 
  window sticker


7" x 5" Decals Available
David Voltz
Cure DBA decal_Voltz.  
Cookbooks Available  
Betty Lightner  
To order online, visit:
cookbook cover  


AmazonSmile Program
You Shop... Amazon Gives!
If you shop on Amazon, please log in using, select Diamond Blackfan Anemia Foundation as your charity, and shop as usual. Amazon will donate .05% of your purchase to the DBAF.
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Raise money for DBAF 
just by searching the web and shopping online!   


Quick Links
The Diamond Blackfan Anemia Foundation (DBAF) is committed to keeping you updated and connected to the entire DBA community. The DBA Foundation is YOUR Foundation!  We encourage you to share your ideas, photos, and stories for our website and upcoming newsletters.  Contact us at
An Overview

pharmacy2.jpgA corticosteroid is a powerful drug that is commonly used to treat many conditions including allergic reactions and inflammation. Although it is not fully understood, corticosteroids also help some DBA patients make more red blood cells. For those patients responding to steroids, the goal is to maintain a hemoglobin level of 9 g/dL or higher without the need for transfusions. When considering the initiation of steroid treatment (often recommended after 1 year of age), it is important to discuss the basic guidelines of this therapy with your physician, taking under consideration the patient's current hematologic status and overall well-being. The main goal in using steroid therapy is to increase red blood cell production while balancing the side effects typical of steroid therapy.

The initial treatment is typically a starting dose of 2 mg/kg/day of prednisone or prednisolone for a trial period of several weeks. With a response to steroids resulting in a rise in hemoglobin, the physician should SLOWLY and carefully lower the initial dose. Over the course of several weeks and months, hemoglobin levels will be monitored and the steroid dose will be adjusted accordingly. Currently the recommended target maintenance dose should not be more than 0.5 mg/kg every day or 1 mg/kg every other day.

Pregnancy and increased growth may warrant an adjustment in steroid dose, and for reasons that are not fully understood, some patients experience unexpected declines in hemoglobin levels and require adjustments to their dose. Colds and other infections may also result in dips in hemoglobin which can be temporary or permanent. Some people experience automatic recovery while others require dose adjustment to increase red blood cell production. Others may requires a blood transfusion to return their hemoglobin to a safe level while waiting for the boosted steroid dose to take effect.

Every DBA patient is different to their response to steroid therapy. Some patients respond to low, seemingly homeopathic doses, some patients have no response, some patients respond only at high doses, and others initially respond but later become refractory. Remission is a possibility for patients on steroids and those on blood transfusion therapy.

Side effects, both short and long-term, are common when taking steroids. It is necessary to be aware, vigilant, and proactive to avoid or minimize the side effects. A low dose of corticosteroids may be adequate to maintain a normal hemoglobin level with few side effects. High doses over a prolonged period can cause serious side effects.  All patients taking steroids should be carefully monitored. Your physician may suggest other treatment options if serious side effects develop.

Possible side effects of short-term use:
WANTED: Amazon Shoppers
Give While Shopping

The Diamond Blackfan Anemia Foundation is participating in the AmazonSmile program. AmazonSmile offers the same wide selection of products, low prices, and convenient shopping features as The important difference is that when customers shop through AmazonSmile (, the AmazonSmile Foundation donates 0.5% of the price of eligible purchases to the charity of your choice!
It simple to do and will not add to the price of your purchase. Visit, select Diamond Blackfan Anemia Foundation as your charity, and let the shopping begin! You can use your existing Amazon account on AmazonSmile. Once you select the Diamond Blackfan Anemia Foundation as your charity, all your shopping visits will benefit the DBAF! It's free and easy. Start your Amazon shopping on AmazonSmile today! 
Where Are You ?
Help Us Keep in Touch

Did you move? Need to add a new address, phone number, email address? Are you a recently diagnosed family?

Please help us keep our records current! Take a moment to visit our website and fill out the registration form. Patient/Family Registration Form 

Help us to reach all our families.
If you are aware of other DBA families in your area, please encourage them to contact the DBA Foundation.

Please note that the Diamond Blackfan Anemia Registry (DBAR) and the Diamond Blackfan Anemia Foundation (DBAF), are not allowed to share your personal information.  It is necessary to register with both the DBAR and the DBAF.

If you have any questions, or to check on the status of your information, contact Dawn at 716.674.2818 or 

Show Us Your Logo

Many thanks to David and Brandi Voltz for initiating this fundraiser to benefit the DBA Foundation. Order yours here:



Here's the challenge: 

We would like to see how many places we can show off our logo!

logoSnap a picture sporting our logo and send us your story. Draw it, print it out, wear it, wave it, tattoo it, carve it, stick it... be creative! Take us to school, on vacation, to the hospital, on a plane, to the game, in your home... anywhere!  Show us your logo!  Send your photos and stories to

Journal Club
An update on gene therapy and DBA
No man is an island,  

Steve Ellis
Steven R. Ellis, PhD
DBAF Research Director

or so sayeth John Donne, a Jacobean poet and preacher.  I'm sure I am not original in my view that what Donne was speaking of with this phrase: most certainly the concept that no person is independent of the influence of others or the influence that they may have on others.

The same could be said of genes.  Genes are not found floating in the nucleus of cells as independent entities.  Nay, they are found as part of chromosomes, 46 of which are found in the nucleus of most cells in our body.  Each chromosome is composed of two strands of DNA winding about one another, one heading left to right and the other right to left (so to speak).  These strands of DNA are composed of deoxyribonucleotides linked to one another through phosphodiester bonds.  There are millions, sometimes hundreds of millions, of deoxyribonucleotide units per strand of DNA in each of our chromosomes.

Within these hundreds of millions of deoxyribonucleotide units is information that codes for the tens of thousands of proteins residing in the human genome.  This information is stored within the sequence of the four-deoxyribonucleotide bases that make up DNA (GATC).  As has been discussed in this forum previously, each of these genes contains additional information (also embodied in the base sequence) needed for the appropriate expression of each gene.  By appropriate, I mean that the gene is expressed in the correct cell type, at a fitting time in development, and in just the right amounts.

When one cell becomes two during cell division, all this genetic information spread across 3.5 billion base pairs of DNA needs to be faithfully copied with one genetic blueprint retained in the original mother cell and the second transmitted to the newly created daughter cell.  This segregation of genetic information between mother and daughter cell is made much easier by having our genes packaged in 46 chromosomes as opposed to having to distribute exact copies of tens of thousands of individual genes between mother and daughter cells during each cell division.

These requisites for faithful transmission of genetic information from one cell to another bring us to this month's Journal Club.  The manuscript I'll be discussing is from the laboratory of Stefan Karlsson, an established investigator in the DBA field who has had a longstanding interest in using gene therapy as a cure for DBA.  The title of the article is "Gene therapy cures the anemia and lethal bone marrow failure in a mouse model for RPS19-deficient Diamond Blackfan anemia"1.  

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