HOW WE ARE MAKING A DIFFERENCE

Parents & Advocates

A key way to ensure positive outcomes for kids with immune  deficiency is for parents and advocates to be prepared, confident and  involved in the process. 
BE GREAT works to create BMT orientation and other programs to prepare parents and advocates.  

Children’s Hospitals

Hospitals focused on pediatric care, such as Children’s National Medical  Center in Washington, D.C., are where it all happens.  
BE GREAT works with children’s hospitals to improve  treatments, programs for parents and access to advance medical  equipment.

Medical Treatment
Newborn screening and improved treatments for immune deficiency are what  can help cure more children. Hospitals and other care providers are the  key to giving kids a chance to live healthy lives.  
BE GREAT works to improve curative treatments and early screening.                

Expectant Families

The generous gift of donating umbilical cord blood can save a life.The logistics of donation can be made easier.   
BE GREAT works with expectant parents and Be The Match who  want to make this gift possible and change someone else’s life.  


BE GREAT Foundation’s programs support children with immune deficiency by:   

  • Expanding diagnostics and research in immunology and transplantation
  • Increasing public cord blood donation
  • Delivering education programs for BMT families
  • Expediting diagnosis time after abnormal newborn screening results 

CENTER FOR DIAGNOSTIC IMMUNOLOGY AND TRANSPLANTATION

Issue:
Children’s National Medical Center in  Washington, DC currently has a diagnostic laboratory focused on  immunology and transplantation.  There is a need to expand its  capability and establish a Center for Diagnostic Immunology and  Transplantation. 


Why it is important:
Broadening Children’s National  capability through a new center will:

  • Increase the number of children that can be treated
  • Expand  diagnosis and research ability
  • Enable the use of the newest technology for improved patient outcomes     


BE GREAT and Children’s National  are investing in kids by finding  the cause of immune deficiency. A Center for Diagnostic Immunology and  Transplantation can lead to:

  • Transformation in the field  for more effective treatments
  • Advanced training for the next generation of immunology transplant physicians
  • Diagnosing babies in-utero to increase their chance of a healthy future 

Children's National Medical Center uniquely has the components needed for a successful center:

  • The only immunology-trained bone marrow transplant physician in the lower mid-Atlantic
  • Genetic testing, infectious disease and other specialists on-staff
  • The physical space to dedicate to a center, if sufficient funding is raised
  • The only exclusive provider of immunologic deficiency  pediatric care in the metropolitan Washington area
  • The only freestanding children’s hospital between Pennsylvania and Georgia

What is needed:

To establish the center,  expansion to the diagnostic laboratory capability is necessary.  This  requires an investment in equipment, personnel, PhD trained specialists  and research staff; so that breakthrough results can be achieved. Goals of the Center for Diagnostic Immunology and Transplantation:

  • Foster new diagnostic techniques and bring them to the clinical setting 
  • Apply unique treatments to these small, rare patient groups and transform the field 
  • Develop treatment protocols to improve outcomes for transplant patients with Severe Immune Diseases 
  • Conduct research to improve diagnostic tools 

What BE GREAT is doing about it
BE GREAT wants  to see the Center for Diagnostic Immunology and Transplantation come to  fruition.   We are sharing Alexander’s experiences to inform others  about the unexpected journey that comes with the diagnosis of an immune  deficiency.  Through our community and corporate events, we are raising  funds to support the center’s development.  This program will directly  impact our belief that every child deserves to be healthy and be great.

How you can help:
If you are interested in finding out more information about this program or becoming involved, contact us.    


Cord Blood Donation Kits

Issue:

Cord blood comes from a newborn baby's umbilical cord and placenta  containing lifesaving cells. Cord blood donation isn't offered as an  option at every U.S. hospital because there are a limited number of  public cord blood banks around the country  It costs a public bank about $1,500 to collect, test, freeze, and  store a single unit of cord blood. The high cost of processing and  storing donated blood and the lack of adequate funding for public cord  blood banks limit the number of public banks. Some states have passed laws to educate expectant mothers about both  public and private cord blood banking. While these laws offer valuable  information to the public, many states don't provide the means for  expectant mothers to easily donate cord blood. As a result, educational  efforts can create a demand that can't be met in many local areas  without additional funds to support storage and collection of cord blood  units.  

Why it is important:

Cord blood cells, like cells from bone marrow, are used in the  treatment of over 70 life-threatening diseases, including immune  deficiency diseases, a wide range of leukemia's, cancers, metabolic  disorders and genetic diseases. To date, more than 10,000 patients  worldwide have been treated using cord blood.    The collection process is safe, easy, and painless for both mother  and baby and does not interfere with delivery. After the baby is born, a  medical professional will  collect the cord blood.  The actual  collection is painless and typically takes three to five minutes.  If the mother meets the eligibility requirements and the cord blood  from the baby is determined to be suitable for transplant, it is stored  at a public cord blood bank and is listed on the National Marrow Donor  Program's Be The Match Registry for use by any patient who may need it.  

What BE GREAT is doing about it:

 Through your support BE GREAT is supporting the public donation  campaign by sponsoring cord blood donation kits to be sent to expectant  families who meet the eligibility requirements for donation and are not  able to deliver their baby at a collection hospital. BE GREAT is  committed to building the cord blood inventory to increase the chances  that more patients like Alexander find their match.
 

How you can help:
If you are interested in finding out more  information about this program or becoming involved, contact us.

BMT FAMILY ORIENTATION

Issue:
The duration of hospital stay for a  Bone Marrow Transplant (BMT) patient and their supportive family members  is varied across the population. At a minimum the stay is at least  three to four weeks.  Through the unexpected journey of immune deficiency, families may not have a full understanding of how to  transition to the new reality of being “at home” in a hospital setting.    

Why it is important:
Preparation is critical, and  it enables parents (and supportive care takers) to increase their  awareness and allow for planning.  Most importantly, preparation creates  the ability to offer patients  comfort during this life saving  journey. Ensuring success in a hospital environment means understanding many things such as:
 

  • Learning hospital terminology and “who is who”
  • Understanding services available to the family
  • Exploring the day in the life of a BMT patient and that of the family care giver
  • Figuring out ways to normalize a very un-normal experience 

s Alexander’s parents reflect on their experience, they were  prepared for the BMT journey through their prior hospital  stays.   However,  not everyone on the BMT journey will  have prior hospital  experience.   As more children are identified as immune deficient  through the newborn screening process, the population of children  admitted into BMT programs without prior infections or hospital stays  will increase.   A lack of hospital experience is likely to be very overwhelming to  families who are already in high-stress situations, such as complex  medical procedures and decisions


What BE GREAT is doing about it:
BE GREAT has  created an Orientation Session for Families embarking on this life  saving journey for their child.  The sessions are facilitated by parents  who have already been on the journey and understand where the family is  in their thoughts and can share the things they wish someone had shared  with them.    BE GREAT Foundation currently facilitates comfort and  knowledge to incoming families to the BMT program at Children’s National  Medical Center.  


How you can help:
If you are interested in finding out more  information about this program or becoming involved, contact us.   Your financial support will help this program continue and expand this program beyond its current reach. 

FAST TRACKING DIAGNOSIS

Issue:

Newborn screening tests are performed when a baby is between 24 hours  and 7 days old, typically before the baby goes home from the hospital.   A few drops of blood are obtained by a nurse from a heel prick on  specially-designed filter paper, to be tested for a panel of  developmental, genetic, and metabolic disorders. The blood is typically  sent to a lab for analysis. Screening tests do not diagnose illnesses,  but rather they identify which babies need additional testing to confirm  or rule out illnesses.


The tests identify whether important  action should be taken during the critical time before symptoms develop.  In the United States, individual states regulate newborn screening, so  the diseases screened for vary considerably from state to state.


On  May 21, 2010, Kathleen Sebelius, Secretary of Health and Human Services  (HHS) announced the addition of Severe Combined Immune Deficiency  (SCID) to the core panel of 29 genetic disorders as part of her  recommendation to states to adopt the national Recommended Uniform  Screening Panel.  In 2011, Maryland, Virginia and the District of  Columbia recommended the addition of SCID to their respective newborn  screen panel.  Through the  expanded newborn screening program happening in many US  states,  parents may be told their baby has an abnormal result for the   SCID condition. Pediatricians need to understand the steps to take,  referrals to offer and information to share with parents as the journey  to diagnosis begins.

Why it is important:

 The survival of SCID patients is greatly enhanced if children are  diagnosed and treated before severe infections take hold.  Babies with  SCID appear healthy at birth, but without early treatment, such as a  bone marrow transplant, these infants cannot survive.  

 

In addition to  SCID, the screening program is able to identify other severe immune  deficiencies in newborns, enabling early diagnosis. The implementation of these screening programs requires the  establishment of a basis of knowledge  for pediatricians to get newborns  babies  connected with specialists for rapid screening, diagnosis  and  identifying a care plan for the child. 

What BE GREAT is doing about it

 BE GREAT is building programs  to work with mid-Atlantic  pediatricians in broadening their knowledge and understanding  of the  steps  necessary when a newborn screening result is abnormal for SCID.   It is critical that all of these babies get on the fast track to  diagnosis. Through your support, this program will continue to  develop as  Maryland, Virginia, and the District of Columbia adopt the Newborn SCID  Screening test.


How you can help:
If you are interested in finding out more  information about this program or becoming involved, contact us.