Bone Marrow Disorders

Question 1

Pure Red Cell Aplasia (PRCA)

Answer 1

Pure red cell aplasia (PRCA) or erythroblastopenia refers to a type of anemia affecting the precursors to red blood cells but not to white blood cells, or platelets.

Characterized by a severe reduction in the number of reticulocytes in the peripheral blood and the virtual absence of erythroid precursors in the bone marrow. Often the bone marrow ceases to produce red blood cells.

Question 2

Pure Red Cell Aplasia (PRCA) Treatment

Answer 2

red cell transfusions for symptomatic anemia
cessation of possible offending drugs
immediate hematologic consultation

Question 3

Myelophthisic Anemia

Answer 3

Myelophthisic anemia (or myelophthisis) is a severe kind of anemia found in some people with diseases that affect the bone marrow.

Myelophythisis refers to the displacement of hemopoietic bone-marrow tissue into the peripheral blood, either by fibrosis, tumors or granulomas.

Question 4

Myelodysplastic syndrome (myelodysplasia or MDS)

Answer 4

Heterogeneous group of hematopoietic stem cell disorders characterized by dysplastic and ineffective blood cell production.

Dysplasia of one or more cell lines is the morphologic hallmark of MDS.

AKA “preleukemia” as it has a variable risk of transformation to acute leukemia

Question 5

Myelodysplastic syndrome (myelodysplasia or MDS) Management

Answer 5

MDS is not known to be curable with conventional Rx such as chemotherapy

Consider transplant in the young, or investigational Rx.

For most patients, treatment is supportive (RBC transfusions, treat infections)

Growth factors, erythropoietin effective in some patients

***Bone marrow transplantation is currently the only potentially curative therapy for MDS patients.

Question 6

Stem cell - Definition

Answer 6

A cell that has the ability to continuously divide and differentiate into various other kind(s) of cells/tissues.

Question 7

Embryonic stem cells

Answer 7

come from a five to six-day-old embryo. They have the ability to form virtually any type of cell found in the human body.

Question 8

Adult stem cells

Answer 8

undifferentiated cells found among specialized or differentiated cells in a tissue or organ after birth. Based on current research they appear to have a more restricted ability to produce different cell types and to self-renew.

Question 9

Embryonic germ cells

Answer 9

are derived from the part of a human embryo or fetus that will ultimately produce eggs or sperm (gametes).

Question 10

Graft Sources

Answer 10

Allogeneic: from another person
Syngeneic: from an identical twin
Autologous: from the patient
Umbilical cord blood

Question 11

Alternative Donors

Answer 11

Matched Unrelated Donors (MUD)
NMDP – Be The Match
Severe GVHD
Higher TRM

Question 12

Haploidentical Donors

Answer 12

From parent, child or sibling
Must have many stem cells to overcome risk of graft rejection
Increased risk of GVHD

Question 13

Allogeneic Transplants

Answer 13

High TRM (30-50%)
Lower relapse rates due to graft versus tumor effects
Graft versus host effects

Question 14

Matched Related Donor (siblings)

Answer 14

25% chance a sibling will be a match

The more siblings a patient has the better chance for a match

Question 15

Autologous Transplant

Answer 15

No evidence of disease in the blood or bone marrow
Transplant related mortality (TRM) lowest with autos (<5%)
Relapse rates are higher depending on the disease
Absence of graft versus host effects

Question 16

Umbilical Cord Blood

Answer 16

Cryopreserved
Small number of stem cells
Higher incidence of engraftment failure
Using more than one unit in adults
Lower risk of GVHD
Degree of matching not as stringent