eMedicine Specialties > Pediatrics: General Medicine > Hematology

Myelofibrosis: Follow-up

Author: J Martin Johnston, MD, Associate Professor of Pediatrics, Mercer University School of Medicine; Director of Pediatric Hematology/Oncology, Backus Children's Hospital; Consulting Oncologist/Hematologist, St Damien's Pediatric Hospital
Contributor Information and Disclosures

Updated: Oct 2, 2009

Follow-up

Further Outpatient Care

  • Supportive care of myelofibrosis (MF) with transfusions (RBC, platelets) is crucial to short-term management. Blood products should be leukodepleted (to decrease the likelihood of human leukocyte antigen [HLA] sensitization) and, ideally, cytomegalovirus (CMV) negative.
  • Prophylaxis against opportunistic infections (eg, fluconazole) may be indicated for some patients with neutropenia.
  • The Mayo Clinic recently described a patient-reported outcomes (PRO) instrument designed specifically to assess quality of life in (adult) patients with myelofibrosis.56

Complications

  • Patients with myelofibrosis but no initial evidence of myelodysplasia or leukemia may ultimately develop either of these conditions.
  • As previously noted, patients can be expected to develop complications secondary to decreased blood counts (eg, anemia, hemorrhage due to thrombocytopenia, opportunistic infections due to leukopenia).
  • Splenomegaly may lead to hypersplenism, thereby worsening pancytopenia.
  • A single case report detailed subcutaneous lymphoma arising in a child with idiopathic myelofibrosis (IMF).57

Prognosis

  • The prognosis depends on the underlying cause of myelofibrosis. With appropriate treatment for rickets, tuberculosis, systemic lupus erythematosus, and other conditions, the myelofibrosis may completely resolve.
  • A retrospective analysis of 203 adult patients with myelofibrosis suggests, in contrast to earlier reports, that myelofibrosis has little adverse effect on engraftment following allogeneic hematopoietic cell transplant.58
  • In adult patients with myelofibrosis, several alternative prognostic scoring systems (PSSs) are available.59,60 Neither the patient's symptoms nor the percentage of circulating blasts is taken into account in the Mayo Clinic PSS (in contrast to other PSSs). A retrospective review of 334 patients with myelofibrosis showed the Mayo Clinic PSS to be more effective than other PSSs in terms of (1) identifying long-lived patients and (2) delineating an intermediate-risk disease category.60 The Mayo PSS assigns a score of 1-4 by allotting 1 point for each of the following:
    • Hemoglobin level more than 10 gm/dL
    • WBC count less than 4 or more than 30 X 109/L
    • Platelet count less than 100 X 109/L
    • Absolute monocyte count equal to or more than 1 X 109/L
  • Idiopathic acute myelofibrosis of childhood (C-AMF) is a fulminant disease. Without effective therapy, life expectancy is typically less than one year. Potentially effective and/or curative treatments include chemotherapy and allogeneic bone marrow transplantation (BMT). Treatment with high-dose corticosteroids or interferon alfa may result in a temporary amelioration of the disease (see Medication). Occasionally, pediatric patients have a more indolent course that is comparable to agnogenic myeloid metaplasia with myelofibrosis (AMMM) in adults. With supportive care alone, they may survive for many years.

Miscellaneous

Medicolegal Pitfalls

  • Splenectomy: Some adult patients with agnogenic myeloid metaplasia with myelofibrosis (AMMM) are candidates for splenectomy, but the surgery-related mortality rate may be as high as 9%. Outcome statistics of splenectomy are not available for pediatric patients. Splenomegaly alone is not an indication for splenectomy.
  • Genetic counseling: Early-onset myelofibrosis (MF) is occasionally inherited in a recessive pattern. Counsel parents about the possibility of a second affected child.
  • Occult or mosaic trisomy 21: Down syndrome is a risk factor for myelofibrosis. Consider chromosomal analysis in any child with onset before age 2 years.
 


More on Myelofibrosis

Overview: Myelofibrosis
Differential Diagnoses & Workup: Myelofibrosis
Treatment & Medication: Myelofibrosis
Follow-up: Myelofibrosis
Multimedia: Myelofibrosis
References
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Further Reading

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Keywords

myelofibrosis, MF, bone marrow fibrosis, myelosclerosis, osteomyelofibrotic syndrome, agnogenic myeloid metaplasia with myelofibrosis, AMMM, acute myelofibrosis of childhood, C-AMF, primary MF, idiopathic MF, IMF, fibrosis of the bone marrow, acute megakaryoblastic leukemia, AMKL, chronic myeloproliferative disorders, clonal hematopoiesis, splenomegaly, erythroblastic peripheral blood smear, myeloid metaplasia, thrombopoiesis, tuberculosis, visceral leishmaniasis, anemia, neutropenia, thrombocytopenia, hepatosplenomegaly, bacterial sepsis, rickets, systemic lupus erythematosus, histiocytosis, acute myeloid leukemia, acute lymphoblastic leukemia, chronic myelogenous leukemia, non-Hodgkin lymphoma, Hodgkin disease, Langerhans cell histiocytosis, sickle cell disease, Fanconi anemia, vitamin D deficiency, osteodystrophy, juvenile rheumatoid arthritis, osteopetrosis, hyperparathyroidism, hypoparathyroidism, pernicious anemia, Gaucher disease, treatment, diagnosis

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Contributor Information and Disclosures

Author

J Martin Johnston, MD, Associate Professor of Pediatrics, Mercer University School of Medicine; Director of Pediatric Hematology/Oncology, Backus Children's Hospital; Consulting Oncologist/Hematologist, St Damien's Pediatric Hospital
J Martin Johnston, MD is a member of the following medical societies: American Society of Pediatric Hematology/Oncology
Disclosure: Nothing to disclose.

Medical Editor

Sharada A Sarnaik, MBBS, Professor of Pediatrics, Wayne State University School of Medicine; Director, Sickle Cell Center, Attending Hematologist/Oncologist, Children's Hospital of Michigan
Sharada A Sarnaik, MBBS is a member of the following medical societies: American Association of Blood Banks, American Association of University Professors, American Society of Hematology, American Society of Pediatric Hematology/Oncology, New York Academy of Sciences, and 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
Disclosure: Pfizer Inc Stock Investment from financial planner; Avanir Pharma Stock Investment from financial planner ; WebMD Salary and stock Employment and investment from financial planner

Managing Editor

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; Assistant Clinical Professor, Department of Pediatrics, Creighton University; 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 Academy of Pediatrics, American Association for Cancer Research, American Federation for Clinical Research, American Society of Hematology, American Society of Pediatric Hematology/Oncology, Council on Medical Student Education in Pediatrics, and Hemophilia and Thrombosis Research Society
Disclosure: Nothing to disclose.

CME Editor

Samuel Gross, MD, Professor Emeritus, Department of Pediatrics, University of Florida; Clinical Professor, Department of Pediatrics, University of North Carolina; 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, Professor of Pediatrics, Oncology and the Cellular and Molecular Medicine Graduate Program, Kimmel Comprehensive Cancer Center at 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 Hematology, and American Society of Pediatric Hematology/Oncology
Disclosure: Nothing to disclose.

 
 
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