eMedicine Specialties > Pediatrics: General Medicine > Oncology

Myelodysplastic Syndrome: Follow-up

Author: Prasad Mathew, MB, BS, DCH, Director, Hemostasis and Hematology Program, Professor of Pediatrics, University of New Mexico
Coauthor(s): Franklin Smith, MD, Marjory J Johnson Endowed Chair, Professor of Pediatrics, Division of Hematology/Oncology, Professor of Pediatrics, University of Cincinnati College of Medicine, Cincinnati Children's Hospital Medical Center; Glenda H Grawe, MD, Assistant Professor, Baylor College of Medicine Department of Pediatrics, Section of Emergency Medicine; Attending Physician, Texas Children's Hospital
Contributor Information and Disclosures

Updated: May 22, 2008

Follow-up

Further Inpatient Care

  • Hospitalization is required to administer some chemotherapeutic agents.
  • Inpatient treatment is required if the patient is undergoing bone marrow transplantation.
  • Children with myelodysplastic syndrome (MDS) should be treated like other patients with neutropenia. They require hospitalization, observation, and intravenous (IV) antibiotics to manage fever.
  • Inpatient admission is required in some locations for transfusion support.

Further Outpatient Care

  • Children should be monitored often because of the propensity of these disorders to transform to acute myeloid leukemia (AML).
  • Patients often require frequent transfusions, and their blood cell counts must be monitored at least monthly.

Inpatient & Outpatient Medications

  • Trimethoprim-sulfamethoxazole should be administered for prophylaxis against Pneumocystis carinii (opportunistic infection).
  • Fluconazole is often administered for prophylaxis against Candida species.
  • Chlorhexidine is recommended to prevent mouth infections.

Transfer

  • Patients should be referred to centers affiliated with major multicenter pediatric oncologic groups.

Complications

  • Infection
    • Severe neutropenia results in life-threatening infection secondary to overgrowth of skin and bowel flora and increased susceptibility to community and hospital pathogens.
    • Patients are extremely susceptible to life-threatening fungal infections.
    • Patients with cytogenetic findings of monosomy 7 or refractory anemia with excess blasts in transition to AML also have poor overall neutrophil function, despite adequate absolute neutrophil counts (ANCs) of more than 1000/µL.
    • Infection, rather than progression to AML, ultimately results in the demise of most patients with MDS.
  • Bleeding
    • Patients often have thrombocytopenia and resultant hemorrhage.
    • Patients require frequent transfusions as marrow is increasingly involved.
  • Anemia and iron overload
    • The inability of marrow to keep up with normal turnover of RBCs results in a frequent need for transfusion. Repeated transfusions may result in iron overload, requiring chelation therapy.
    • In rare circumstances, patients respond to splenectomy.
    • Iron overload is observed most often in adults with MDS related to transfusions over a prolonged course.

Prognosis

  • Patients with Down syndrome and MDS respond best to treatment, whereas those with MDS due to previous therapy with alkylating agents fare the worst. As noted above, patients without Down syndrome who undergo allogeneic HSCT have the best outcome, despite transplant-related mortality.  
  • Continued multicenter trials are needed with further elucidation of biologic markers to best classify MDS in childhood. Studies of medications such as azacitidine, decitabine, and lenalidomide should be undertaken to elucidate the efficacy in the pediatric population.
  • Until recently, most of the prognostic factors in MDS, such as those used in the IPSS, the Bournemouth score, and others, were based on data from adult patients.
    • In adults, factors that have had prognostic significance for survival and progression to AML include bone marrow morphology, myeloblast percentage in the bone marrow, the appearance of the bone marrow on biopsy findings, number of cytopenias, cytogenetic abnormalities in bone marrow, age, and blood lactate dehydrogenase levels.
    • The only factor that has consistently had prognostic significance in children with MDS is cytogenetic abnormality, notably monosomy 7. 
    • Researchers from Japan, the United Kingdom, and the European Working Group on MDS in Childhood have all concluded that the IPSS is of limited value in children. Investigators from Japan and the United Kingdom found that only the IPSS karyotype group had significant prognostic value in terms of overall survival.
  • In the United States, one prospective study (CCG 2891) provided results about the effects of AML-based therapy in children with MDS.9 Of 1096 patients enrolled, 90 had MDS classified according to the FAB classification. Overall survival at 6 years was 29% ±12 for patients with MDS and 31% ±26 for those with JMML treated in the CCG 2891. These outcomes were worse than those of patients who had antecedent MDS and who were treated in the AML phase (50% ±25) or those of patients with de novo AML (45% ±3). Nonsignificant differences in 6-year survival were observed between patients with juvenile myelomonocytic leukemia (JMML) and MDS.
  • In recent reports, 5-year EFS rates in patients with Down syndrome and MDS and/or AML were in excess of 80%. These rates were largely because of reductions in treatment-related deaths from 30-40% in the early 1990s to around 10% in recent Berlin-Frankfurt-Münster (BFM), Nordic Society of Paediatric Haematology and Oncology (NOPHO), and Medical Research Council studies.

Patient Education

  • Families must be educated about signs and symptoms of infection, anemia, and thrombocytopenia.
  • Many patients require placement of a central venous catheter, and their families need to learn how to care for the line.
 


More on Myelodysplastic Syndrome

Overview: Myelodysplastic Syndrome
Differential Diagnoses & Workup: Myelodysplastic Syndrome
Treatment & Medication: Myelodysplastic Syndrome
Follow-up: Myelodysplastic Syndrome
References
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Further Reading

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Keywords

myelodysplastic syndrome, MDS, MDS, chronic myelomonocytic leukemia, CMML, clonal hemopathy, juvenile chronic myeloid leukemia, JCML, juvenile myelomonocytic leukemia, JMML, monosomy 7, oligoblastic leukemia, preleukemia, refractory anemia, RA, smoldering acute leukemia, acute myelogenous leukemia, acute myeloid leukemia, AML, adult-type MDS, a-MDS, refractory anemia with ringed sideroblasts, RARS, refractory anemia with excess blasts, RAEB, refractory anemia with excess blasts in transition to AML, RAEBT

cytopenia, preleukemia, hematopoietic stem cell transplantation, HSCT, 5q- syndrome, 5q deletion syndrome, infantile monosomy 7, myeloproliferative disorders, bone marrow dysfunction, neurofibromatosis type 1, NF1, cytopenia, short stature, obesity, gonadal failure, hypothyroidism, cataracts, bone marrow failure, lymphadenopathy, therapy-related MDS, Down syndrome, myeloid leukemia of Down syndrome, ML-DS, pancreatic insufficiency, Fanconi anemia, Kostmann syndrome, Diamond-Blackfan anemia, dyskeratosis congenita

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Contributor Information and Disclosures

Author

Prasad Mathew, MB, BS, DCH, Director, Hemostasis and Hematology Program, Professor of Pediatrics, University of New Mexico
Prasad Mathew, MB, BS, DCH is a member of the following medical societies: American Society of Hematology
Disclosure: Nothing to disclose.

Coauthor(s)

Franklin Smith, MD, Marjory J Johnson Endowed Chair, Professor of Pediatrics, Division of Hematology/Oncology, Professor of Pediatrics, University of Cincinnati College of Medicine, Cincinnati Children's Hospital Medical Center
Disclosure: Nothing to disclose.

Glenda H Grawe, MD, Assistant Professor, Baylor College of Medicine Department of Pediatrics, Section of Emergency Medicine; Attending Physician, Texas Children's Hospital
Glenda H Grawe, MD is a member of the following medical societies: American Academy of Pediatrics, American College of Emergency Physicians, Harris County Medical Society, Minnesota Medical Association, National Association of EMS Physicians, and Texas Pediatric Society
Disclosure: Draeger Honoraria Review panel membership

Medical Editor

Kathleen Sakamoto, MD, Professor, Department of Pediatrics, Division of Hematology-Oncology and Pathology and Laboratory Medicine, Mattel Children's Hospital, David Geffen School of Medicine, University of California at Los Angeles
Kathleen Sakamoto, MD 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, and Western Society for Pediatric Research
Disclosure: Nothing to disclose.

Pharmacy Editor

Mary L Windle, PharmD, Adjunct Assistant Professor, University of Nebraska Medical Center College of Pharmacy, Pharmacy Editor, eMedicine.com, Inc
Disclosure: Pfizer Inc Stock Investment from broker recommendation; Avanir Pharma Stock Investment from broker recommendation

Managing Editor

Timothy P Cripe, MD, PhD, Associate Professor of Pediatric Hematology/Oncology, University of Cincinnati; Director, Translational Research Trials Office, Department of Pediatrics, Cincinnati Children's Hospital Medical Center
Timothy P Cripe, MD, PhD is a member of the following medical societies: American Association for the Advancement of Science, American Society of Hematology, and American Society of Pediatric Hematology/Oncology
Disclosure: Nothing to disclose.

CME Editor

Samuel Gross, MD, Professor Emeritus, Department of Pediatrics, University of Florida, Clinical Professor, Department of Pediatrics, UNC, Adjunct Professor, Department of Pediatrics, Duke University
Samuel Gross, MD is a member of the following medical societies: American Association for Cancer Research, American Society for Blood and Marrow Transplantation, American Society of Clinical Oncology, American Society of Hematology, and Society for Pediatric Research
Disclosure: Nothing to disclose.

Chief Editor

Robert J Arceci, MD, PhD, King Fahd Professor of Pediatric Oncology, Department of Oncology, Division of Pediatric Oncology, Johns Hopkins University School of Medicine
Robert J Arceci, MD, PhD is a member of the following medical societies: American Association for Cancer Research, American Association for the Advancement of Science, American Pediatric Society, American Society of Clinical Oncology, American Society of Hematology, and American Society of Pediatric Hematology/Oncology
Disclosure: Nothing to disclose.

 
 
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