Other Conditions We Treat
The Cincinnati Children’s Bone Marrow Transplantation team has developed significant expertise in performing stem cell transplantation for children with many other kinds of rare diseases that do not fall in the categories of malignancy, immune system disorders, HLH, hemoglobinopathies or bone marrow failure syndromes. Some examples of additional diagnoses that we have transplanted:
Leukodystrophies. Inherited diseases hallmarked by dysfunctional metabolism of important fatty acids. This results in abnormal deposition of myelin (the protection around nerves), poor brain development and death. Examples we have transplanted include:
- Krabbe’s disease
- Metachromatic dystrophy
- Mucopolysaccharidoses such as Hurler’s syndrome. Metabolic disorders caused by absent or dysfunctional lysosomal (cellular) enzymes needed to break down molecules called glycosaminoglycans. This results in the inappropriate deposition of these molecules in various tissues of the body.
- Osteopetrosis. An inherited skeletal dysplasia or disease hallmarked by dysfunctional bone deposition and breakdown.
- Porphyria. A disorder of hemoglobin metabolism such as congenital erythropoietic porphyria (Gunther’s disease) with significant skin and organ injury.
Bone marrow transplantation can increase the risk of pain and discomfort in our patients. We have a dedicated and experienced team of doctors and nurse practitioners ready to provide sensitive care for this concern.
Learn about the Pain Management Service for children established by Cincinnati Children’s.