Cord Blood Cord Blood Diseases and Treatments 1 — Questions and Answers
Question 1: Which form of childhood leukemia was one of the first diseases successfully treated with cord blood transplantation?
- Chronic lymphocytic leukemia (CLL)
- Acute lymphoblastic leukemia (ALL) (Correct answer)
- Hairy cell leukemia
- Adult T-cell leukemia
Correct answer: Acute lymphoblastic leukemia (ALL)
Acute lymphoblastic leukemia (ALL), particularly in children, was among the first malignancies treated with allogeneic cord blood transplantation as a curative approach.
Question 2: How does cord blood transplantation help patients with sickle cell disease?
- It replaces the sickling red blood cells with donor-derived normal red blood cells (Correct answer)
- It corrects the gene directly in existing cells
- It provides pain medications to manage crises
- It removes the spleen to reduce complications
Correct answer: It replaces the sickling red blood cells with donor-derived normal red blood cells
Cord blood transplantation replaces the patient's defective hematopoietic stem cells with healthy donor cells, leading to production of normal hemoglobin and a potential cure for sickle cell disease.
Question 3: What is beta-thalassemia major and how does cord blood transplantation address it?
- A platelet disorder treated with transfusion only
- A hemoglobin disorder caused by absent or reduced beta-globin production, cured by replacing defective stem cells with healthy donor cord blood (Correct answer)
- A clotting factor deficiency treated with infusions
- A white blood cell cancer treated with chemotherapy only
Correct answer: A hemoglobin disorder caused by absent or reduced beta-globin production, cured by replacing defective stem cells with healthy donor cord blood
Beta-thalassemia major is a severe inherited hemoglobin disorder caused by mutations in the HBB gene; allogeneic cord blood transplantation can replace defective stem cells with healthy donor cells, potentially curing the disease.
Question 4: What is Fanconi anemia and why is cord blood transplantation preferred for its treatment?
- An acquired anemia treated with iron supplements; cord blood is not preferred
- An inherited bone marrow failure syndrome with DNA repair defects; cord blood is preferred due to its lower GVHD risk and tolerance of HLA mismatches (Correct answer)
- A nutritional deficiency; cord blood provides missing proteins
- A viral infection cured by antivirals
Correct answer: An inherited bone marrow failure syndrome with DNA repair defects; cord blood is preferred due to its lower GVHD risk and tolerance of HLA mismatches
Fanconi anemia is an inherited DNA repair disorder causing bone marrow failure and cancer predisposition; cord blood transplant is preferred because patients are hypersensitive to conditioning agents and benefit from the lower GVHD rates of cord blood.
Question 5: Which metabolic storage disorder affecting the brain is treated with cord blood transplantation to halt neurological progression?
- Phenylketonuria (PKU)
- Krabbe disease (globoid cell leukodystrophy) (Correct answer)
- Celiac disease
- Wilson's disease
Correct answer: Krabbe disease (globoid cell leukodystrophy)
Krabbe disease is a fatal lysosomal storage disorder; cord blood transplantation in newborns before symptom onset can halt neurological deterioration by providing healthy enzyme-producing cells.
Question 6: What neurological condition has shown encouraging results in clinical trials using autologous cord blood infusion?
- Multiple sclerosis
- Cerebral palsy (Correct answer)
- Amyotrophic lateral sclerosis (ALS)
- Alzheimer's disease
Correct answer: Cerebral palsy
Clinical trials, including studies at Duke University, have shown that autologous cord blood infusion may improve motor function in children with cerebral palsy, likely through neuroprotective mechanisms.
Cord Blood Cord Blood Diseases and Treatments 1
Which form of childhood leukemia was one of the first diseases successfully treated with cord blood transplantation?