Issue: # 37 
April 2014 
In This Issue
A DBA Superstar
DBAF Provides Gap Funding
DBAF Thanks DBA Nurse, Ellen Muir
Commit to a Cure
Show Us Your Logo
Journal Club
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Superstar = AUDREY!!

We are pleased to tell you the story of adorable Audrey Nethery and her incredible family. Often times, families are intimidated to host a fundraiser. Here is the story of one very special family from Louisville, KY, written by Audrey's grandmother, Sharon.



Approximately nine months ago a very good friend, Shannon, came to me and  said, "I want to do a benefit in honor of Audrey for the DBA Foundation." This was something I had always thought about doing, but never thought I could do it.  She convinced me that we should at least try, so we started planning - first the date.  A woman from our church donated a Masonic Hall and so the journey began.


The next few months we received numerous gift cards by asking and giving out the brochure that we made to our local businesses.  We always had our brochures with us and everywhere we ate or shopped, we always asked for donations.  To our surprise, the generosity of the businesses was amazing, but if someone said no we did not get discouraged, we just kept asking... every time.  We were able to make two baskets over $600 worth of gift cards to raffle at the benefit.  We even had around ten or more cards to give as door prizes. Some businesses even donated their own baskets.  In all, by the time the benefit date was here,we had 15 baskets for raffles, 12 silent auction items and the door prizes (all donated).


We ordered the food, hired a DJ and then waited for the big night.  We were nervous and scared, but to our amazement, on the night of the benefit, there was standing room only!!! No one complained; everyone was there to support a good cause and enjoy a great evening. When the event was over, Shannon and I got into the car to leave. Without a word, we turned and gave each other a "high five." We knew we had done well. When we got home, we started to add up the checks and cash.  We are proud to say that we are sending the DBA Foundation a check for $10,000.00 with this letter. It would never had happened if we had not had the support of family & friends, church family, and our work family.


But most of all, it happened because of a true friend who gave me the confidence and support to do something that I had never done before.


Scott, Audrey's dad added, "It was amazing and humbling to feel the love and support of everyone that cares about us. We are in awe of how good it feels to have our entire community support the DBA Foundation. We are grateful to be able to help fund research for our daughter and all DBA patients. It was amazing!"

The DBA Foundation thanks Sharon, Shannon, the entire Nethery family and all their family and friends that helped make this a great event! THANK YOU!

The Netherys appearing on a local TV show to promote their event benefiting the DBA Foundation.
Upcoming Events


Annual Strike Out DBA 

Bowling Fundraiser
April 26, 2014
Grand Haven, MI
Tammi Lanore


Cleveland HUGS Fundraiser for DBA

August 16, 2014

Wesley Memorial United Methodist Church

Cleveland, TN


Robert Lee


Friends of DBA Golf Outing  September 15, 2014
Fox Meadow Golf Club
Medina, OH 
Jim & Carol Mancuso


Friends of DBA 5K / 1 mile Walk 
October 4, 2014
Fox Meadow Golf Club
Medina, OH 
Jim & Carol Mancuso

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)
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In memory of...
Holiday giving...
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5" x 5" Decals Available
Dawn Baumgardner 
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7" x 5" Decals Available
David Voltz
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Cookbooks Available  
Betty Lightner  
To order online, visit:
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Award Given to Dr. Harvey Lodish

The Diamond Blackfan Anemia Foundation (DBAF) is proud to announce the funding of another important research project. Through the dedication, hard work, and generosity of our families, friends, and supporters, the DBAF was able to award $21,281 to Dr. Harvey Lodish's project entitled, "High-throughput screening identifies many novel potential therapies for Diamond Blackfan Anemia."  The esteemed Dr. Lodish provided the following summary of his lab's very exciting work and the significance of the Diamond Blackfan Anemia Foundation's gap funding.

Xiaofei Gao, Dr. Harvey Lodish, and Sherry Lee


High-throughput screening identifies many novel potential therapies for Diamond-Blackfan Anemia.


Sherry Lee, Xiaofei Gao, Lingbo Zhang, Lina Prak, Shilpa Hattangadi, and Harvey Lodish  
Whitehead Institute and Departments of Biology and Biological Engineering, Massachusetts Institute of Technology, Cambridge MA 02142  

Many Diamond Blackfan Anemia (DBA) patients respond to treatment with the steroid prednisone with increases in red cells, but continuous use of this and other corticosteroids causes severe side effects. We need additional drugs to treat this disease, and one way of proceeding is to screen existing drugs and related chemicals for their ability to stimulate red cell production.

Supported by generous grants from the U S Department of Defense, we screened a collection of over 2000 tested and approved therapeutic compounds for those that can stimulate mouse red cell production in culture. We obtained over 40 potential "hits" and then rescreened all of them in a new human cell culture system we developed. Ten drugs approved for human use stimulated human red cell production in this system at a developmental stage that suggests they may be useful in treating DBA.

DBAF Thanks DBA Nurse, Ellen Muir
Announcement from the DBAR


The DBA Foundation thanks Ellen Muir for her many years of dedication and care. We wish her the very best in her new position. Thank you, Ellen. You will be missed!


Dear DBA Community: 

As often happens, people move on in their professional lives, so sadly we must report to all of you that Ellen Muir, DBA Nurse, is moving on to a new nursing position in our Division of Hematology/Oncology and Stem Cell Transplantation. As you all know Ellen has been working with the Diamond Blackfan Anemia Registry since 2006. She has been a wonderful asset for the DBAR and I am sure for all of you. She has accepted a new position to run our clinical area at the Cohen Children's Medical Center. Thus, if you come for a DBA Surveillance and Awareness Program visit, you will still see Ellen. We will all miss our DBA Nurse - but we will have Ellen at the outpatient area daily.


We will all try our best to respond to all your phone calls and e-mails in a timely way. But, of course, Ellen is irreplaceable in so many ways.


We also would like the opportunity to thank Ellen publicly for all her work in our "DBA World". As a friend and colleague she is one of the most dedicated people we know. She is an amazing, loving person and will bring the best to all in her future endeavors.


We are actively searching for another fine nurse to add to our team. Ellen will help us choose her successor and will help us train her (or him).

Please send your inquiries regarding clinical matters to any of us in the meantime. We will let you know once we have chosen a new DBA Nurse. Our number remains 516-562-1504 or 516-562-1505. Our toll-free numbers are 1-888-884-DBAR (3227) and 1-877-DBA-NURS (1-877-322-6877).


Your DBA Team,

Adrianna Vlachos, MD
Jeffrey M. Lipton, MD, PhD
Johnson M. Liu, MD
Eva Atsidaftos, MA, CCRC

Become A Monthly Donor
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Over the past two years, The Diamond Blackfan Anemia Foundation donation_jar_give.jpg

has reached out to our families asking for their support. Thank you to the families that have responded with their personal donations, and thank you to the families that have made a monthly pledge. We are so pleased that more and more families are making a monthly commitment to the DBAF. From $20/month to $200/month, these monthly donations are easy to set up through your bank or through our website using a credit card or PayPal. Of course, a monthly check can also be mailed to the DBAF. Please join the many other families that have made this commitment.


Your generous support is appreciated and will help all DBA families. Please visit our website for donation information.

Since 1994, your donations and fundraising efforts have allowed the Diamond Blackfan Anemia Foundation (DBAF) to provide funding for major research projects that have resulted in the establishment of the DBA National Registry (DBAR), the discovery of defective "DBA genes," a better understanding of DBA on both the molecular and genetic levels, advances in creating animal models, novel approaches to less toxic treatments, and promising advances towards successful therapeutic cures. We have sponsored, supported and cosponsored international scientific meetings, have sponsored national family conferences and retreats, and have provided our families with informational materials and support. 


While we are proud of our accomplishments, we realize we must continue to work diligently to fulfill our mission. We need your help. Please consider partnering with us to help find a cure for DBA. I am happy to answer any questions you may have regarding fundraising efforts or the DBAF.  


Thank you for your careful consideration. Please commit to find a cure!

Dawn Baumgardner
Executive Director

Show Us Your Logo

Looks like DBA has a "Mystery Sticker-er!" There are reports of our logo stickers showing up in many different places, across many different states. This photo was taken in Edgewood, Maryland. 

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

And They're Off

Steve Ellis
Steven R. Ellis, PhD
DBAF Research Director


It's the last week in April. The grass is green, the sky a deep blue, birds chirping and rabbits hopping to and fro in search of early season plantings. This time of year has a special meaning for those of us here in Louisville, Kentucky.  Pollen!  Well, yes: we were just given the dubious distinction of America's #1 city for allergy suffers, and not in a good way. More importantly for Louisvillians, is that the last week in April transitions to the first Saturday in May and the Kentucky Derby! The city is, and has been, in a flutter with events spreading over three weeks in what is known as the Kentucky Derby Festival. These events include art shows, balloon and steamboat races, a marathon, a parade, the Kentucky Oakes, Derby Eve festivities (loaded with celebrities), and of course, the Derby itself. 


I, like many others in the city, have been caught up in these events if for no other reason than the extra time it takes to get from here to there with all the traffic congestion and re-routing that comes with masses of people coming together for a good time. An obvious question at this point is what has all this got to do with readers of this column living their lives well outside of Louisville and its immediate surroundings: nothing really, other than to state that this will be a relatively short column covering a relatively short paper1.


While the paper may be short in length, it is nonetheless of critical importance to transfusion-dependent DBA patients and their families.  Dr. Thomas Coates spearheaded these studies.  If his name sounds familiar, it should, as I mentioned this work in my summary of the DBA ICC held in 2012. Since the work presented at ICC conferences is unpublished, my descriptions have to be painted in broad strokes to avoid releasing privileged information and allowing investigators the freedom to share work fresh from the lab bench without fear of having their work jeopardized by premature discloser. This tempered approach ends when a paper gets published.


The studies from the Coates laboratory investigate iron loading in different tissues in various cohorts of patients receiving regular transfusions. The authors used Magnetic Resonance Imaging (MRI) as a non-invasive method to monitor iron in the liver, pancreas, and heart in chronically transfused patients. The bottom line from these studies you've heard before: patients with DBA load iron differently than many other patients: doing so rapidly and early in the course of receiving transfusion therapy. The manuscript provides the details on which this statement is based.  


One hundred and twenty five children were included in this study.  Of these, 17 had DBA.  Other diseases represented were sickle cell, thalassemia major, congenital dyserythropoietic anemia (CDA), pyruvate kinase deficiency, and others including children being transfused after cancer treatments.  The initial iron measurements were carried out as early as ~22 months in the DBA cohort with a mean age of first MRI at 3.8 years.  While all patients showed the presence of liver iron at an early age, patients with DBA were notable in this study for highest percentage of patients with the presence of pancreatic and cardiac iron at the time of the first MRI, including a patient presenting with cardiac iron at the age of two.  


These studies provide a detailed documentation of iron loading in DBA patients relative to other patients receiving chronic transfusions.  They provide the numbers supporting the largely anecdotal views that DBA patients load iron differently than other patients receiving chronic transfusions; including both rapid and early accumulation of tissue iron in the pancreas and heart, in addition to the liver.  They build on an earlier report on Italian DBA patients that focused predominantly on liver iron, which supported the same conclusion2.  


While the Coates study does not provide the mechanism by which DBA patients load iron differently from many other patient cohorts receiving chronic transfusion, they do underscore the need for regular surveillance of tissue iron in DBA patients and aggressive chelation therapy; points made time and time again at Camp Sunshine and other DBA venues.   


With respect to monitoring iron levels in the body, Dr. Coates has an additional, more recent publication on the use of ferritin as a measure of tissue iron3. His conclusions on using ferritin as a measure of tissue are similar to points of emphasis made by Dr. Lawrence Wolfe on several occasions.  I think it is worthwhile to quote Dr. Coates directly on this subject, "While ferritin is a convenient measure of iron status; ferritin trends were unable to predict changes in LIC in individual patients. Ferritin trends need to be interpreted with caution and confirmed by direct measurement of LIC." 

LIC = Liver Iron Concentrations


1. Berdoukas, V., Nord, A., Carson, S., Puliyel, M., Hofstra, T., Wood, J., and Coates, T.D. (2013). Tissue iron evaluation in chronically transfused children shows significant levels of iron loading at a very young age. American journal of hematology 88, E283-285.

2. Roggero, S., Quarello, P., Vinciguerra, T., Longo, F., Piga, A., and Ramenghi, U. (2009). Severe iron overload in Blackfan-Diamond anemia: a case-control study. American journal of hematology 84, 729-732.

3. Puliyel, M., Sposto, R., Berdoukas, V.A., Hofstra, T.C., Nord, A., Carson, S., Wood, J., and Coates, T.D. (2014). Ferritin trends do not predict changes in total body iron in patients with transfusional iron overload. American journal of hematology 89, 391-394.

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