Chronic granulomatous disease (CGD) is an immune deficiency where the neutrophils (a type of white blood cell that kills bacteria and fungi) do no work properly. Some patients with CGD have neutrophils that do not work at all, whereas others have neutrophils that work partially, but not normally. In the past (over 20 years ago), most patients with CGD were managed with antibiotics and antifungal medications alone. As the science of blood and marrow transplant (BMT) improved, some patients with CGD started to receive a BMT. It remained controversial whether patients with CGD should receive a BMT or medical management alone (antibiotics, antifungals, and other treatments that do not include BMT). It appears that some patients clearly benefit from BMT whereas others may receive less benefit.
6903 is trying to better define what the role of BMT compared to medical management of CGD is. In particular, what are the outcomes of BMT versus medical management alone, why do some patients clearly benefit from BMT, and what are the long-term outcomes of both approaches. Researchers are interested in how CGD patients who have no neutrophil function may differ from CGD patients with some neutrophil function, how the types of infections and inflammatory complications of CGD impact on survival and how BMT may improve these complications. There are also questions as to how the types of bacteria (called the microbiome) found in the gastrointestinal tract (colon, large intestine) of CGD patients influences certain inflammatory complications (such as colitis), and how BMT changes the microbiome in CGD patients. All of this will help doctors in the future to better treat patients with CGD.
6903 enrolls patients who meet the criteria for diagnosis of CGD. Not only is 6903 enrolling patients from all North American PIDTC sites, 3 centers from Europe (London, Newcastle, Zurich) are also enrolling patients on this study. Speak to your doctor to see if you / your child are eligible. This is NOT a clinical research trial. There are NO experimental treatments and the study is not dictating how you / your child’s doctor should treat you. It is only asking for data around how well you / your child has done with whatever treatments your doctor feels are necessary for the best medical care of you / your child. The 6903 study includes a retrospective (looking back into the past), cross-sectional (one time collection of information and/or research testing) and a prospective (looking from today and into the future) component to the 6903 study. These are known as longitudinal studies ie, they look at information of patient over time. Persons with CGD who were born 1988 to the present day are eligible, regardless of whether they received a BMT (as long as the BMT was after 1995) or medical therapy only. Patients who are newly diagnosed with CGD can also be enrolled and followed longitudinally (over time), to determine their outcome from the choice of therapy that is made. An important component of 6903 study is the ‘cross sectional’ study, where patients with more than 3 years of follow-up after transplant or diagnosis are asked to provide additional research blood work and information is gathered regarding long-term transplant outcomes such as infections, graft-versus-host disease, autoimmune diseases, and quality of life. In addition, the 6903 study will request stool samples from patients to look at how certain bacteria found in the gut (called the microbiome) affect complications of CGD such as gastrointestinal disease. This will allow PIDTC researchers to better understand the outcomes of different therapeutic approaches and to best design new treatments and clinical trials in the future for children with CGD.
Participating Sites
Alfred I. duPont Hospital for Children/Nemours
1600 Rockland Road
Wilmington, Delaware, United States, 19803
https://www.nemours.org/locations/wilmington-ai-dupont-childrens-hospital.html
Contact:
Emi
Caywood
American Family Children's Hospital
1111 Highland Ave, 4103 WIMR
Madison, Wisconsin, United States, 53705-2275
https://www.uwhealth.org/locations/american-family-childrens-hospital-169
Contact:
Ken
DeSantes
Ann & Robert H. Lurie Children's Hospital of Chicago
225 E Chicago Avenue
Chicago, Illinois, United States, 60611
https://www.luriechildrens.org
Contact:
Sonali
Chaudhury
Baylor College of Medicine/Texas Children's Hospital
1 Baylor Plaza
Houston, Texas, United States, 77030
https://www.bcm.edu/
Contact:
Imelda Celine
Hanson
Boston Children's Hospital
300 Longwood Avenue
Boston, Massachusetts, United States, 02115
https://www.childrenshospital.org/
Contact:
Craig
Platt
craig.platt@childrens.harvard.edu
Susan Prockop Malika KapadiaCardinal Glennon Children's Hospital
1465 S Grand Boulevard
Saint Louis, Missouri, United States, 63104
https://www.ssmhealth.com/locations/cardinal-glennon-childrens-hospital
Children's & Women's Health Centre of British Columbia
4500 Oak Street
Vancouver, British Columbia, Canada, V6H 3N1
http://www.bcwomens.ca/
Contact:
Stuart
Turvey
Children's Healthcare of Atlanta: AFLAC Cancer Center
1405 Clifton Road NE, 4th Floor, Tower 1
Atlanta, Georgia, United States, 30322
https://www.choa.org
Contact:
Shanmuganathan
Chandrakasan
Children's Hospital Colorado
13123 E 16th Avenue, B115
Aurora, Colorado, United States, 80045
https://www.childrenscolorado.org/locations/anschutz-medical-campus-aurora/
Contact:
Hesham
Eissa
Children's Hospital Los Angeles
4650 Sunset Boulevard
Los Angeles, California, United States, 90027
https://www.chla.org/
Contact:
Neena
Kapoor
Children's Hospital of Wisconsin
8915 W Connell Court
Milwaukee, Wisconsin, United States, 53226
https://childrenswi.org/location-directory/locations/hospitals/milwaukee-hospital
Contact:
John M.
Routes
Children's Hospital/LSUHSC, New Orleans
200 Henry Clay Avenue, Suite 4109
New Orleans, Louisiana, United States, 70118
https://www.chnola.org
Contact:
Lolie
Yu
Children's National Medical Center
111 Michigan Ave NW
Washington, District of Columbia, United States, 20010
https://childrensnational.org
Contact:
Blachy Davila
Saldana
Duke University Medical Center
2301 Erwin Road
Durham, North Carolina, United States, 27710
https://www.dukehealth.org/locations/duke-university-medical-center
Contact:
Rebecca H.
Buckley,
MD
Hackensack University Medical Center
30 Prospect Ave
Hackensack, New Jersey, United States, 07601
https://www.hackensackmeridianhealth.org/
Contact:
Alfred
Gillio
Johns Hopkins All Children's Hospital
601 5th Street South, 3rd Floor
St. Petersburg, Florida, United States, 33701
https://www.jhu.edu/
Contact:
Deepak
Chellapandian
Lucile Packard Children's Hospital Stanford
450 Serra Mall
Stanford, California, United States, 94305
https://www.stanford.edu/
Contact:
Ami
Shah
Maria Fareri Children's Hospital
100 Woods Road
Valhalla, New York, United States, 10595
https://www.mariafarerichildrens.org
Contact:
Mitchell S.
Cairo,
MD
Mayo Clinic Rochester
200 First Street SW
Rochester, Minnesota, United States, 55905
https://www.mayoclinic.org/patient-visitor-guide/minnesota
Contact:
Anvi
Joshi
Memorial Sloan Kettering Cancer Center
1275 York Avenue
New York, New York, United States, 10065
https://www.mskcc.org/
Contact:
Joseph
Oved
National Institute of Allergy and Infectious Diseases
5601 Fishers Lane, MSC 9806
Bethesda, Maryland, United States, 20892
https://www.niaid.nih.gov/
Contact:
Harry
Malech,
MD
Nationwide Children's Hospital
700 Childrens Drive
Columbus, Ohio, United States, 43205
https://www.nationwidechildrens.org/
Contact:
Peter
Mustillo
peter.mustillo@nationwidechildrens.org
Hemalatha Rangarajan Roshini AbrahamOregon Health & Science University
3181 S.W. Sam Jackson Park Road
Portland, Oregon, United States, 97239-3098
https://www.ohsu.edu
Contact:
Evan
Shereck
Phoenix Children's Hospital: Center for Cancer and Blood Disorders
1919 E Thomas Road
Phoenix, Arizona, United States, 85016
https://appointments.phoenixchildrens.com/Appointments/FindPatient
Contact:
Roberta H.
Adams
St. Jude Children's Research Hospital
262 Danny Thomas Place
Memphis, Tennessee, United States, 38105
https://www.stjude.org/
Contact:
Ewelina
Mamcarz
The Children's Hospital of Alabama
ACC 512, 1600 7th Avenue South
Birmingham, Alabama, United States, 35233
https://www.uab.edu/home/
Contact:
Fred
Goldman
University of Rochester
500 Joseph C. Wilson Boulevard
Rochester, New York, United States, 14627
https://www.rochester.edu/
Contact:
Jeffrey
Andolina
jeffrey_andolina@urmc.rochester.edu
Katherine Tuttle Geoffrey Weinberg