Bone Marrow Failure

Question 1

What is pancytopenia

Answer 1

it includes low rbc wbc and plts.

Question 2

what is bone marrow failure definition

Answer 2

pancytopenia due to failulre of bm to produce blood cells.

Question 3

what are thee symptoms of bm failure

Answer 3

anemia, difficulty breathing, chest pain and fatigue

Question 4

what are symtpoms of leukopenia/neutropenia

Answer 4

fever, infxn, mouth sores

Question 5

what are symtpoms of thrombocytopenia

Answer 5

bleeding

Question 6

what are things that may lead to hypercellular marrow?

Answer 6

hematologic malignancies, leukemias,myeloma, lymphoma, carcinoma, storage disoreders, myelodysplastic syndromes, b12 or folate deficiency

Question 7

what are things leadings to hypocellular marrow

Answer 7

congential (fanconis anemia)

acquired (idiopathic, myelodysplastic syndrome, drugs radiation,viruses)

Question 8

what is aplastic anemia?

Answer 8

sever, life-threatening syndrome in which production of rbcs, wbcs and plts has failed. may occur in all age groups
charaxterized by peripheral pancytopenia and accompanied by a hypocellular bone marrow

Question 9

what is the pathophysiology of aplastic anemia?

Answer 9

primary defect is reduction in or depletion of hematopoeitic precursor stem cells. this is what leads to peripheral pancytopenia.

Question 10

what three problems can lead to aplastic anemia in terms of pathways?

Answer 10

failure of self-renewal, proliferation or differentiation, or no cytokines and gf from environment

Question 11

what is fanconi’s anemia

Answer 11

usually symptomatic around age 5 and has progressive bm hypoplasia. congenital defects such as skin hyperpigmentation and small stature are also seen in affected individuals

Question 12

what is familial aplastic anemia

Answer 12

a subset of fanconi’s in which congenital defects are absent

Question 13

what are viral infxns that can lead to aplastic anemia?

Answer 13

mononucleosis, infxs hepatitis, parvovirus, and cytomegalovirus infxns as well as tuberculosis

Question 14

what are lab findings of aplastic anemia?

Answer 14

severe pancytopenia with relative lymphocytosis, normochromic rbcs, mild to moderate anisocytosis and poikilocytosis, decreased retic count, hypocellular bone marrow with more than 70% yellow marrow

Question 15

how do you treat aplastic anemia?

Answer 15

withdrawal of offending agent
transfusions, antibiotics
immunosuppressive regimns
hsct

Question 16

what is pure red cell aplasia?

Answer 16

pure red cell aplasia is characterized by a selective decrease in erythroid precursor cells in bm. wbcs and plts are unaffected.

Question 17

what are myelodysplastic syndromes?

Answer 17

primary, neopalstic stem cell disorders that ten to terminate in acute leukemia. normally bm is normocellualar or hyperceull. with evidnce of qualitiative abnormaliites in one or more cell lines resutling in ineffective erythropoiesis

Question 18

what is a common histo sighting in mds?

Answer 18

ringed sideroblasts

Question 19

When do myelodysplastic syndromes become Acute Myelogenous Leukemia?

Answer 19

when the blast count rises about 20% for who and 30% for fba

Question 20

What are the goals of treatment of mds?

Answer 20

control symptoms, improve quality of life, improve overall survival, and decrease progression to acute myelogenous leukemia

Question 21

what is the mainstay of treatment of myelodysplastic syndromes?

Answer 21

supportive care with blood products and hematopoeitic growth factors (epo)

Question 22

what chemotherapy agent is used with myelodysplastic syndromes?

Answer 22

methylating agent 5-azacytidine and decitabine (they decrease the need for blood transfusions and retard the progression of mds to aml and increase survival)

Question 23

what was approved in 2005 for the treatment of mds with the 5q syndrome?

Answer 23

lenalidomide

Question 24

what in general can be down for mds patients that are younger and more severely affected patients?

Answer 24

hsct

Question 25

What is autologous stem cell transplant?

Answer 25

It is when stem cells are collected from the patient, stored in a freezer, and reused within that patient later following a high dose of chemotherapy.

Question 26

what are the advantages of allogeneic stem cell transplant?

Answer 26

they can be used when recipient’s bone marrow fails such as in aplastic anemia and myelodysplastic syndrome. when recipient has a certain disease such as leukemia and lymphoma, the donor cells can attack these tumor cells via graft versus disease effect to prevent relapse of the tumor.

Question 27

What are disadvantages of hsct?

Answer 27

higher risk of chemotherapy related complications
higher risk of infxn compared to auto sct
risk of gvhd

Question 28

what is myeloablative hsct?

Answer 28

bm is ablated with dose-levels of chemo that cause minimal injury to other tissues. the chemo or irradiation given immediately prior to a translant is called the condition of preparative regimen, prior to infusion of hsc.

Question 29

what is non-myeloablative hsct?

Answer 29

newer tx approach using lowerdoses of chemotherapy and radiation which are too low to eradicate all of the bm of a recipient. goal is to prevent donor cells from being rejected by recipient. also called reduced intensity allogeneic transplant.

Question 30

why would you use non-myeloablative hsct?

Answer 30

1. it is less intense, so there are less complications associated with typical chemotherapy.
2. leaving some of your original stem cells can lead to the beneficial effect of graft vs disease.

Question 31

what are the complications of infxns due to hsct?

Answer 31

bm transplant usually requires destruction of your own cells, leading to low numbers of immune cells to fight infxn
immunosuppressive agents employed in allogeneic transplants for the prevention of tx of gvhd further increase risk of infxn
patient must be revaccinated because they lose their acquired immunity.

Question 32

what are additional complications due to hsct?

Answer 32

veno-occlusive disease
mucositis
gvhd (acute and chronic)

Question 33

how do you treat gvhd?

Answer 33

prevent of acute with cyclosporine and methotrexate to supress immune response
tx with high dose of steroids

Question 34

when is graft versus tumor disease beneficial?

Answer 34

when it’s a slowly progressing tumor, chronic leukemia, low-grade lymphoma, and some cases multiple myeloma. less effectve in rapidly growing acute leukemias.