Which Statements Are True?
Statements a, b, c, and e are true; statement d is false.
Statement-by-Statement Analysis
a) Lenalidomide is used in multiple myeloma: TRUE
- Lenalidomide combined with dexamethasone is an established treatment for relapsed or refractory multiple myeloma, demonstrating 58-59% response rates in phase III trials 1.
- This regimen provides a treatment option alongside bortezomib and thalidomide-based regimens for relapsed/refractory disease 1.
- The standard dosing is lenalidomide 25 mg/day orally in combination with dexamethasone, continued until disease progression 1.
b) Cytarabine (Ara-C) is used in acute leukemia: TRUE
- Cytarabine is a cornerstone agent in acute myeloid leukemia (AML) treatment, used in multiple regimens 2.
- The NCCN guidelines specifically include cytarabine-based induction regimens such as ATRA + cytarabine (AIDA regimen) for acute promyelocytic leukemia 2.
- For higher-risk MDS and AML, combinations of cytarabine with idarubicin, fludarabine, or topotecan are standard regimens with equivalent efficacy 2.
- Low-dose cytarabine (20 mg/m²/day for 14-21 days every 4 weeks) is also used as an alternative treatment option in certain MDS patients 2.
c) Lenalidomide is used for myelodysplastic syndromes: TRUE
- Lenalidomide is FDA-approved for low-risk MDS with deletion 5q cytogenetic abnormality 1.
- The ESMO guidelines recommend lenalidomide for anemia in lower-risk MDS with del(5q), achieving 60-65% response rates with median RBC transfusion independence of 2-2.5 years 2.
- The recommended starting dose is 10 mg/day orally for 3 weeks every 4 weeks 2.
- Cytogenetic responses occur in 50-75% of patients (including 30-45% complete cytogenetic responses) 2.
- Lenalidomide should be the drug of choice for patients with low and intermediate-1 risk MDS with chromosome 5q31 deletion 1.
- In the EU, lenalidomide is approved specifically for lower-risk MDS with del(5q) and RBC transfusion dependence after failure or ineligibility to erythropoietin-stimulating agents 2.
d) Vitamin B12 is the treatment of choice for immune thrombocytopenic purpura: FALSE
- This statement is completely incorrect. Vitamin B12 is used to treat B12-deficiency megaloblastic anemia, not immune thrombocytopenic purpura (ITP) 2.
- ITP is an autoimmune disorder causing platelet destruction and requires treatments such as corticosteroids, intravenous immunoglobulin, or thrombopoietin receptor agonists—not vitamin supplementation.
- Vitamin B12 deficiency causes macrocytic anemia with low reticulocytes, not thrombocytopenia 2.
e) Folic acid is the treatment of choice for folate-deficiency megaloblastic anemia: TRUE
- The FDA label explicitly states that folic acid is effective in the treatment of megaloblastic anemias due to folic acid deficiency 3.
- This includes megaloblastic anemias seen in tropical or nontropical sprue and anemias of nutritional origin, pregnancy, infancy, or childhood 3.
- Folate deficiency is characterized by macrocytic anemia with normal or low reticulocytes and occurs with increased folate requirements (pregnancy, hemolysis, chronic myeloid leukemia) 2.
Critical Caveats
For lenalidomide in MDS:
- Close monitoring for grade 3-4 neutropenia and thrombocytopenia is essential, occurring in ~60% of patients during the first weeks of treatment 2.
- TP53 mutations (found in ~20% of del(5q) MDS) confer resistance to lenalidomide and higher risk of AML progression, requiring intensified surveillance 2.
- Dose reduction is frequently required due to myelosuppression 1.
For cytarabine in acute leukemia:
- AML-like intensive chemotherapy with cytarabine has limited indication in higher-risk MDS patients, particularly those with unfavorable karyotype who show few complete remissions and shorter CR duration 2.
- Low-dose cytarabine was found significantly inferior to azacitidine in terms of response and survival in a randomized phase III study 2.