Pediatric Myelodysplasia Follow-up

Updated: Oct 25, 2018
  • Author: Natalia Dixon, MD; Chief Editor: Robert J Arceci, MD, PhD  more...
  • Print
Follow-up

Further Outpatient Care

Outpatient follow-up care depends on the degree of anemia and thrombocytopenia. Close follow-up is warranted, as progression to frank AML can occur over weeks to months.

Transfusion support is now manageable in the outpatient setting. Packed RBCs and platelets need to be leukofiltered and irradiated. Donor exposure to platelets should be minimized with pheresis and single-donor products whenever possible. This minimizes the risk of development of alloimmunization and the risk of the patient becoming refractory to transfusions.

Next:

Further Inpatient Care

Inpatient admission for patients with myelodysplasia syndrome (MDS) is usually for treatment of fever during periods of neutropenia. These episodes require aggressive evaluation for a source of infection and empiric coverage with broad-spectrum antibiotics against gram-negative rods. Blood and urine should be cultured for bacteria and for fungus, depending on the duration of symptoms.

Inpatient care at a designated center is also needed for stem cell transplantation.

Previous
Next:

Inpatient & Outpatient Medications

Patients are often placed on Pneumocystis carinii pneumonia (PCP) prophylaxis because of their degree of immunosuppression. Trimethoprim-sulfamethoxazole (Bactrim, Septra) is commonly used on a 3-times-per-week schedule. In patients allergic to Bactrim or in cases of Bactrim-related myelosuppression, oral atovaquone or aerosolized pentamidine is effective on a monthly schedule.

Previous
Next:

Transfer

Patients should be referred to centers with established stem cell transplant programs and experience in treating myelodysplasia syndrome and other hematologic malignancies.

Previous
Next:

Complications

Infection

Patients with myelodysplasia syndrome may have increased risk for infection due to depressed granulocyte number and function. Even in cases of normal neutrophil number, neutrophils may exhibit decreased myeloperoxidase and microbicidal activity. Granulocytes may exhibit poor adhesion, chemotaxis, phagocytosis, and decreased microbicidal activity. Patients are extremely susceptible to life-threatening gram-negative rod and fungal infections.

Bleeding

Patients often have thrombocytopenia and resultant hemorrhage. Platelet dysfunction may occur in myelodysplasia syndrome. Patients require frequent transfusions as the bone marrow becomes increasingly hypoplastic.

Anemia

In rare circumstances, iron overload is a complication of chronic red blood cell transfusions and may necessitate iron chelation therapy.

Previous
Next:

Prognosis

Findings associated with a poorer prognosis in childhood myelodysplasia syndrome include refractory anemia with excess blasts (RAEB) and refractory anemia with excess blasts in transformation (RAEBT). Age younger than 2 years and hemoglobin F levels greater than 10% have proven in several series to be unfavorable features in patients. However, this is because most patients with these features have JMML, a disease that has proven refractory to all therapies tried to date, except for early allogeneic HSCT, which can be curative in some cases. Patients with myelodysplasia syndrome and major chromosomal abnormalities, such as monosomy 7, have a dismal prognosis unless they proceed to allogeneic HSCT.

Karyotype is the most important factor for progression to advanced myelodysplasia syndrome. Patients with monosomy 7 have significantly higher probability of progression than patients with other chromosomal abnormalities or normal karyotypes. [19] Spontaneous disappearance of monosomy 7 and cytopenia have been reported but remains a rare event. [38] In contrast, patients with trisomy 8, Down Syndrome or normal karyotype may experience a long stable course of their disease.

Previous
Next:

Patient Education

Patient education should relate to prevention and treatment of complications of thrombocytopenia and neutropenia, as outlined in Treatment. In cases in which patients have a central venous access device, parents must be educated with regard to its care.

Previous