Development and Validation of a Symptom Scale for Children With Chronic Graft-Versus-Host Disease
| Status: | Completed |
|---|---|
| Conditions: | Orthopedic, Hematology |
| Therapuetic Areas: | Hematology, Orthopedics / Podiatry |
| Healthy: | No |
| Age Range: | 5 - 18 |
| Updated: | 4/2/2016 |
| Start Date: | January 2008 |
| End Date: | July 2010 |
| Contact: | David A Jacobsohn, MD |
| Email: | djacobsohn@childrensmemorial.org |
| Phone: | 773-880-3694 |
Development and Validation of a Symptoms Scale for Children With Chronic Graft-Versus-Host Disease
Chronic Graft-versus-Host Disease (cGVHD) is an important cause of morbidity and mortality
in patients undergoing allogeneic bone marrow transplantation. cGVHD usually occurs after
100 days following transplantation and develops in 20-60% of transplant recipients. The
incidence of cGVHD varies depending on the age of the marrow recipient, the use of sibling
or unrealted donor bone marrow, teh use of unmanipulated T cell-depleted bone marrow, and
perhaps other factors. Clinically, cGVHD is characterized by multi-system disease, which
frequently mimics the clinical features of autoimmune diseases. The manifestations include
skin changes (lichenoid and sclerodermatous changes, changes in pigmentation, loss of
accessory structures such as hair, dystrophic nails, and rash), joint contractures, severe
cramping, hepatic dysfunctions, sicca syndrome, obstructive lung disease, esophageal
dysmotility, weight loss, polyserositis, immunodeficiency, and autoantibodies including
anti-nuclear antibody, anti-erythrocyte antibodies, and anti-platelet antibodies.
in patients undergoing allogeneic bone marrow transplantation. cGVHD usually occurs after
100 days following transplantation and develops in 20-60% of transplant recipients. The
incidence of cGVHD varies depending on the age of the marrow recipient, the use of sibling
or unrealted donor bone marrow, teh use of unmanipulated T cell-depleted bone marrow, and
perhaps other factors. Clinically, cGVHD is characterized by multi-system disease, which
frequently mimics the clinical features of autoimmune diseases. The manifestations include
skin changes (lichenoid and sclerodermatous changes, changes in pigmentation, loss of
accessory structures such as hair, dystrophic nails, and rash), joint contractures, severe
cramping, hepatic dysfunctions, sicca syndrome, obstructive lung disease, esophageal
dysmotility, weight loss, polyserositis, immunodeficiency, and autoantibodies including
anti-nuclear antibody, anti-erythrocyte antibodies, and anti-platelet antibodies.
A large number of children with cGVHD have to deal with many years of a disfiguring and
painful chronic illness with the side effects of long term steroid use. The number of stem
cell transplants done in children is only growing given that we are now transplanting
patients with a variety of nonmalignant disorders and given the use of alternative donor
sources. The broad categories of limited and extensive cGVHD are recognized by clinicans,
but are not particularly useful in clinical practice. Since cGVHD may involve almost every
organ system adn since cGVHD constitutes a waxing and waning nature, cGVHD makes clinical
management very difficult and complicated. Currently, there is a symptoms scale used in the
adult population for measuring symptom burden for adults with cGVHD. This scale is called
the "Lee Symptoms Scale". The purpose of this project is to develop a scale that is similar
in design to the Lee Scale, but it is specifically designed to measure the burden of cGVHD
in the pediatric population
painful chronic illness with the side effects of long term steroid use. The number of stem
cell transplants done in children is only growing given that we are now transplanting
patients with a variety of nonmalignant disorders and given the use of alternative donor
sources. The broad categories of limited and extensive cGVHD are recognized by clinicans,
but are not particularly useful in clinical practice. Since cGVHD may involve almost every
organ system adn since cGVHD constitutes a waxing and waning nature, cGVHD makes clinical
management very difficult and complicated. Currently, there is a symptoms scale used in the
adult population for measuring symptom burden for adults with cGVHD. This scale is called
the "Lee Symptoms Scale". The purpose of this project is to develop a scale that is similar
in design to the Lee Scale, but it is specifically designed to measure the burden of cGVHD
in the pediatric population
Inclusion Criteria:
- 5-18 years of age
- Prior allogeneic Stem Cell Transplant, with any graft source, donor type, and GVHD
prophylaxis allowed
- Clinical diagnosis of cGVHD
- Need for systemic treatment, defined as any medication or intervention delivered
- No evidence of primary disease relapse
- Signed, informed consent, and if applicable, adolescent assent
Exclusion Criteria:
- Inability to give signed informed consent
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