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.

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.

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.

Transfer

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

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.

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.^[21]Spontaneous disappearance of monosomy 7 and cytopenia have been reported but remains a rare event.^[42]In contrast, patients with trisomy 8, Down Syndrome or normal karyotype may experience a long stable course of their disease.

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.

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Morphological findings of refractory cytopenia of childhood.
Morphological findings of hypoplastic refractory cytopenia of childhood and aplastic anemia of childhood.

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Author

Natalia Dixon, MD Assistant Professor, Department of Pediatrics, Division of Hematology and Oncology, Wake Forest University School of Medicine

Natalia Dixon, MD is a member of the following medical societies: American Academy of Pediatrics, American Society of Hematology, American Society of Pediatric Hematology/Oncology, Children's Oncology Group

Disclosure: Nothing to disclose.

Coauthor(s)

Sharon M Castellino, MD, MSc, FAAP Associate Professor, Department of Pediatrics, Section Pediatric Hematology-Oncology, Wake Forest University Health Sciences

Sharon M Castellino, MD, MSc, FAAP is a member of the following medical societies: Alpha Omega Alpha, American Academy of Pediatrics, American Society of Hematology, American Society of Pediatric Hematology/Oncology

Disclosure: Nothing to disclose.

Scott C Howard, MD Professor and Associate Dean for Research, University of Tennessee Health Science Center College of Medicine

Scott C Howard, MD is a member of the following medical societies: American Society of Clinical Oncology, American Society of Hematology, American Society of Pediatric Hematology/Oncology, Central American Pediatric Hematology-Oncology Association, Columbian Pediatric Hematology/Oncology Society, International Society of Paediatric Oncology

Disclosure: Nothing to disclose.

Specialty Editor Board

Mary L Windle, PharmD Adjunct Associate Professor, University of Nebraska Medical Center College of Pharmacy; Editor-in-Chief, Medscape Drug Reference

Disclosure: Nothing to disclose.

James L Harper, MD Associate Professor, Department of Pediatrics, Division of Hematology/Oncology and Bone Marrow Transplantation, Associate Chairman for Education, Department of Pediatrics, University of Nebraska Medical Center; Associate Clinical Professor, Department of Pediatrics, Creighton University School of Medicine; Director, Continuing Medical Education, Children's Memorial Hospital; Pediatric Director, Nebraska Regional Hemophilia Treatment Center

James L Harper, MD is a member of the following medical societies: American Society of Pediatric Hematology/Oncology, American Federation for Clinical Research, Council on Medical Student Education in Pediatrics, Hemophilia and Thrombosis Research Society, American Academy of Pediatrics, American Association for Cancer Research, American Society of Hematology

Disclosure: Nothing to disclose.

Chief Editor

Jennifer Reikes Willert, MD Associate Clinical Professor, Department of Pediatrics, Division of Pediatric Hematology/Oncology, Section of Stem Cell Transplantation, Stanford University Medical Center, Lucile Packard Children's Hospital

Jennifer Reikes Willert, MD is a member of the following medical societies: American Academy of Pediatrics, American Society for Blood and Marrow Transplantation, American Society of Hematology, American Society of Pediatric Hematology/Oncology, Children's Oncology Group

Disclosure: Nothing to disclose.

Additional Contributors

Sharada A Sarnaik, MBBS Professor of Pediatrics, Wayne State University School of Medicine; Director, Sickle Cell Center, Associate Hematologist/Oncologist, Children's Hospital of Michigan

Sharada A Sarnaik, MBBS is a member of the following medical societies: American Society of Hematology, American Society of Pediatric Hematology/Oncology, New York Academy of Sciences, Society for Pediatric Research, Children's Oncology Group, American Academy of Pediatrics, Midwest Society for Pediatric Research

Disclosure: Nothing to disclose.

Acknowledgements

The authors and editors of eMedicine gratefully acknowledge the contributions of previous authors Carmen Arkansas, MD, and Glenda Grawe, MD, to the development and writing of this article.