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Cecil's Ch. 45- Hematopoiesis &
Hematopoietic Failure Questions & Verified
Answers Updated 2025-2026 Graded A
5-Azacytidine - ANS DNA methyltransferase inhibitor; delays the time to
leukemic transformations in 2/3 of patients with transfusion-dependent
MDS, decreases transfusion requirements, and improves quality of life
compared with transfusion support alone; only drug shown to significantly
prolong overall survival of MDS patients, thus it is the standard of care for
transfusion-dependent MDS patients
5q- syndrome - ANS MDS with an isolated deletion in the long arm of
chromosome 5; patients are predominantly older women with a refractory
macrocytic anemi, normal or elevated platelet counts, and overall better
clinical outcomes compared to MDS with 7p-, monosomy 7, or trisomy 8
Alemtuzumab - ANSa humanized monoclonal antibody directed against the
CD52 protein found on lymphocytes and which has efficacy in other
autoimmune diseases, has been as effective as rabbit ATG and
cyclosporine in relapsed and refractory severe AA
Allogeneic stem cell transplantation - ANSabnormally functioning
hematopoietic bone marrow is eradicated and replaced with normal bone
marrow or stem cells from a compatible source (i.e. related or unrelated
donor); high-dose chemotherapy is used to destroy the patient's bone
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marrow, followed by infusion of new stem cells that engraft and restore
normal hematopoiesis
Aplastic anemia (AA) - ANSa rare disorder characterized by pancytopenia
with a markedly hypocellular bone marrow; occurs predominantly in young
adults (20-25 years old) and older adults (60-65 years old)
Autologous stem cell transplantation - ANSthe patient's bone marrow or
peripheral blood stem cells (PBSCs) are collected during remission after
high-dose chemotherapy or G-CSF administration and are cryopreserved,
thawed, and reinfused; incurs higher risk of relapse as a result of reinfusion
of a stem cell product that may remain contaminated with tumor cells
Benefits of PBSC transplantation - ANSdecreased neutrophil recovery time,
lower transfusion requirements, fewer inpatient hospital days, similar rates
of acute GVHD and long-term survival outcomes as traditional marrow-
transplants
Bone marrow cellularity in patients with AA - ANS5-15%, with increased fat
accumulation and few or no hematopoietic cells
Cause of 5q- syndrome - ANSdefects in ribosomal protein function,
specifically the ribosomal subunit protein RPS14
CD34 - ANScell differentiation antigen, expressed by HSC
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Cecil's Ch. 45- Hematopoiesis &
Hematopoietic Failure Questions & Verified
Answers Updated 2025-2026 Graded A
5-Azacytidine - ANS DNA methyltransferase inhibitor; delays the time to
leukemic transformations in 2/3 of patients with transfusion-dependent
MDS, decreases transfusion requirements, and improves quality of life
compared with transfusion support alone; only drug shown to significantly
prolong overall survival of MDS patients, thus it is the standard of care for
transfusion-dependent MDS patients
5q- syndrome - ANS MDS with an isolated deletion in the long arm of
chromosome 5; patients are predominantly older women with a refractory
macrocytic anemi, normal or elevated platelet counts, and overall better
clinical outcomes compared to MDS with 7p-, monosomy 7, or trisomy 8
Alemtuzumab - ANSa humanized monoclonal antibody directed against the
CD52 protein found on lymphocytes and which has efficacy in other
autoimmune diseases, has been as effective as rabbit ATG and
cyclosporine in relapsed and refractory severe AA
Allogeneic stem cell transplantation - ANSabnormally functioning
hematopoietic bone marrow is eradicated and replaced with normal bone
marrow or stem cells from a compatible source (i.e. related or unrelated
donor); high-dose chemotherapy is used to destroy the patient's bone
1
, 2
marrow, followed by infusion of new stem cells that engraft and restore
normal hematopoiesis
Aplastic anemia (AA) - ANSa rare disorder characterized by pancytopenia
with a markedly hypocellular bone marrow; occurs predominantly in young
adults (20-25 years old) and older adults (60-65 years old)
Autologous stem cell transplantation - ANSthe patient's bone marrow or
peripheral blood stem cells (PBSCs) are collected during remission after
high-dose chemotherapy or G-CSF administration and are cryopreserved,
thawed, and reinfused; incurs higher risk of relapse as a result of reinfusion
of a stem cell product that may remain contaminated with tumor cells
Benefits of PBSC transplantation - ANSdecreased neutrophil recovery time,
lower transfusion requirements, fewer inpatient hospital days, similar rates
of acute GVHD and long-term survival outcomes as traditional marrow-
transplants
Bone marrow cellularity in patients with AA - ANS5-15%, with increased fat
accumulation and few or no hematopoietic cells
Cause of 5q- syndrome - ANSdefects in ribosomal protein function,
specifically the ribosomal subunit protein RPS14
CD34 - ANScell differentiation antigen, expressed by HSC
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