eMedicine Specialties > Pediatrics: General Medicine > Oncology

Myelodysplastic Syndrome

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

Introduction

Background

Myelodysplastic syndrome (MDS) in childhood encompasses a diverse group of bone marrow disorders that share a common clonal defect of stem cells and that result in ineffective hematopoiesis with dysplastic changes in the marrow. These disorders are characterized by one or more cytopenias despite a relatively hypercellular bone marrow. MDS disorders are referred to as preleukemias because of their tendency to transform into acute myeloid leukemia (AML). 

MDS is rare in childhood and may have a rapidly progressive course with an extremely poor prognosis without hematopoietic stem cell transplantation (HSCT). The disease can arise in a previously healthy child; in this case, it is referred to as de novo or primary MDS. MDS may develop in a child with a known predisposition; this is secondary MDS. The disease is most common in adults, especially elderly people, and the course varies, ranging from an acute, rapidly fatal illness to a chronic, indolent illness.

MDS is classified into groups according to findings on peripheral blood smears, bone marrow histology, and clinical examination. Notable controversy surrounds classification based on a systematic evaluation of frequency, outcomes, and treatment difficulty. Most accepted systems are modification of the classification of adult MDS the French-American-British (FAB) group proposed.1  Children with MDS whose disease fit in these classes are often considered to have adult-type MDS in current studies.

Types in the FAB system are the following:

  • Refractory anemia (RA)
  • RA with ringed sideroblasts (RARS)
  • RA with excess blasts (RAEB; 5-20% marrow blasts)
  • RAEB in transition to AML (RAEBT; 20-30% marrow blasts)
An exception to the FAB system is the classification of chronic myelomonocytic leukemia (CMML). Numerous children fit this criterion; however, their peripheral blood smears often reveal more than 5% blasts. In addition, most children who otherwise fulfill criteria for CMML have an extremely poor prognosis compared with adults with CMML, who have a course more prolonged than that observed in other forms of MDS. 

CMML, as it occurs in adults, is extremely rare in pediatric populations. Because of differences between adults and children, this entity has been referred to as juvenile myelomonocytic leukemia (JMML) or juvenile chronic myelogenous leukemia (JCML). The currently preferred term is JMML. Because JMML is a separate entity from MDS, it is not discussed in detail in this article. MDS in children and adults differs in other ways; for example, RARS is exceedingly rare in children, and constitutional abnormalities are observed in many children but few adults. 

One of the criticisms of the FAB system is that it does not include the prognostic implications of cytogenetic findings or other biologic features. Of note are 5q- syndrome (5q deletion syndrome), monosomy 7 syndrome, and infantile monosomy 7. Monosomy 7 is most often associated with JMML, and as many as 30% of children with JMML have a deletion of all or part of chromosome 7. Although this finding imparts some prognostic value concerning morbidity, its contribution in predicting mortality is controversial. 

In an attempt to better characterize these disorders and incorporate cytogenetic information, the World Health Organization (WHO) described an alternate classification scheme for MDS.2 As described below, the WHO classification eliminated the RAEBT category and added an unclassified category. The WHO classification is as follows:

  • RA or RARS (erythroid dysplasia only, marrow blasts <5%)
  • RA with multilineage dysplasia (blasts <5%)
  • 5q- syndrome (blasts <5%, no other genetic abnormalities)
  • RAEB (blasts 5-20%)
  • MDS unclassified (does not fit into above groups) 
Another classification schema directed toward MDS in childhood, mainly adapted by the European community, included MDS (refractory cytopenia, RAEB and RAEBT), JMML, and Down syndrome–specific diseases. The changing classification schemes and continuing controversies reflect a limited understanding of MDS. An adequate scheme is likely to be devised only after detailed comprehension of MDS at its genetic, biologic, and clinical levels is attained. MDS and myeloproliferative disorders were included for the first time in the international classification of childhood cancers in 2005.3

Pathophysiology

MDS is a clonal disorder. Aberration occurs in a stem cell that can give rise to multiple lineages. This event explains the presence of multiple derangements observed in the bone marrow that involve several cell lineages. As the affected cell lines continue to divide and to provide the marrow with dysplastic cells, bone marrow dysfunction becomes apparent. This state may persist until a clone undergoes further transformation to leukemia and the marrow becomes fibrotic and aplastic. As an alternative, the clone may progressively deteriorate, and the appearance of marrow may return to normal as healthy stem cells repopulate it. The natural progression of MDS is, thus, a function of an abnormal clone leading to progressive loss of marrow function, transformation to AML, or spontaneous remission.  

The observation of cytogenetic abnormalities, most specifically monosomy 7 and neurofibromatosis type 1 (NF1) genetic mutations, support the theory that cell dysregulation occurs in a multihit fashion. In monosomy 7, a genetic predisposition and a later loss of a critical region on chromosome 7 that encodes a suspected tumor suppressor gene is suggested to set the stage for proliferation of an abnormal clone. Loss of the chromosome may occur during an embryonic period in hematopoietic stem cells or may result from cytotoxic therapy.  

In patients with NF1, function of the NF1 gene product, neurofibronin (a glutamyl transpeptidase [GTPase]) is decreased, resulting in the loss of negative feedback RAS. Therefore, RAS is constitutively active in NF1. Farnesyltransferase inhibitors are able to inhibit activated RAS by preventing the required farnesylation reaction from occurring. Murine experiments suggest that RAS mutations disturb hemopoietic differentiation and lead to a proliferative advantage of hematopoietic precursor cells, ineffective erythropoiesis, and anemia.

Monosomy 7 occurs in approximately 30% of primary childhood MDS cases and in about 50% of therapy-related MDS cases.

The 5q- syndrome is considered a distinct MDS subtype, characterized by 5q-, less than 5% bone marrow blasts, normal or elevated platelet counts, longer survival, and an increased response to lenalidomide (Revelmid). Although 5q- is occasionally reported in children, the typical 5q- syndrome has not been reported.

Frequency

United States

The distribution of FAB classifications in adult populations is as follows:

  • RA - 38.4%
  • RARS - 11.5%
  • RAEB - 15%
  • RAEBT - 3.9%
  • CMML - 31.2%
In the pediatric population, aggressive forms such as RAEB and RAEBT are more common than RA or RARS. 

The epidemiologic literature on childhood MDS is sparse. Factors for this lack of information include the following:

  • A widely accepted classification is lacking.
  • Patients with indolent forms of the disease may not be referred to a tertiary center. This practice may result in a bias among institution-based studies toward the aggressive forms.
  • Cancer registries do not generally register patients with MDS.
  • The incidence is not well known. In one of the earliest reports, MDS or preleukemia was reported in 17% of childhood AMLs (2.9% of all children with leukemia).4 Other studies confirmed that a preleukemic phase precedes AML in about 12-20% of children with AML.5 These studies were based on referrals for suspected AML and did not include the less advanced cases of MDS.

International

The few population-based studies have given conflicting data about the incidence of MDS. Population-based data from Denmark and Canada (British Columbia) showed that MDS and JMML represented 6% of all hematologic malignancies in children, corresponding to annual incidences of 1.8 and 1.2 cases per million children and adolescents aged 0-14 years, respectively.6  

A similar rate of MDS and JMML (7.7% in combination with childhood leukemia) was found in Japan, where therapy-related MDS represents 23% of all cases. 

In England, the incidence is reported to be 0.5 case per million population, which accounts for 1.1% of childhood hematologic malignancies. The exclusion of secondary MDS may only partly explain the relatively low incidence in the United Kingdom. The incidence in elderly people is 89 per 100,000 population.

Mortality/Morbidity

  • The prognosis for pediatric patients with MDS is poor without HSCT. The most common cause of death is cytopenia.
  • One study that included adults showed that the prognosis for Japanese patients with RA was significantly more favorable than that of German patients (median survival 175 mo vs 40 mo, P <.01).7 This result suggests an ethnic variation in survival between Asian and Caucasian populations. Furthermore, the cumulative risk of acute leukemia evolution was significantly lower in Japanese patients than in German patients.
  • Most long-term complications are related to myeloablative therapy with stem cell rescue. Sequelae include short stature, obesity, gonadal failure, hypothyroidism, and cataracts.

Race

  • Data from the Children's Cancer Group showed that 75% of patients are Caucasian, 8.5% are Hispanic, 8% are African American, 3.5% are Asian, and 5% are of unknown race or ethnicity.8  
  • Most studies have been conducted in countries with predominately Caucasian populations. Therefore, results may not reflection the true racial distribution.
  • The incidence for each race has not been reported.

Sex

  • Combined data from 290 patients with mainly primary MDS showed a nearly-equal sex distribution.
  • In patients with adult-type MDS such as RA, RAEB, and RAEBT, the male-to-female ratio is 1.2:1.

Age

MDS occurs in people of all ages.

  • For adult-type MDS, the median age is 5-8 years.
  • Data from about 290 children with primary MDS showed a median age of 6.8 years.

Clinical

History

  • Children have a history consistent with bone marrow failure. Their history and presentation are similar to those of children with leukemia.
  • The interval between the onset of symptoms and diagnosis is 0-23 months, with a median of 2 months.
  • Patients may be asymptomatic, and the condition may be discovered when a routine CBC count is obtained.
  • Other symptoms include the following:
    • Fatigue
    • Systemic infection (bacterial or fungal)
    • Prolonged fever
    • Bruising, bleeding

Physical

  • Children have findings consistent with bone marrow failure. The presentation may resemble that of acute leukemia.
  • General appearances range from well to constitutional wasting.
  • Pallor and fatigue due to anemia may be present.
  • Hepatosplenomegaly predominates in juvenile myelomonocytic leukemia (JMML).
  • Lymphadenopathy is present in 40-76% of patients with JMML but is present in less than 10% of patients with adult-type myelodysplastic syndrome (MDS).
  • About 30% of patients with JMML have a diffuse erythematous, maculopapular rash.

Causes

MDS may be primary or secondary. Children with primary MDS may have an underlying but unknown genetic defect that predispose them to develop MDS at a young age. Secondary MDS occurs in patients after chemotherapy or radiation therapy (therapy-related MDS) or in patients with inherited bone marrow failure disorders, acquired aplastic anemia, or familial MDS. Therefore, the distinction between primary MDS and secondary MDS may become arbitrary.

  • Approximately 20% of children have an underlying congenital anomaly or syndrome associated with chromosomal abnormalities.
  • MDS and acute myeloid leukemia (AML) in Down syndrome are closely linked; the biologic and clinical features are distinct from the diseases observed in children without Down syndrome. In the proposed WHO classification, MDS and AML in Down syndrome are recognized as a single specific entity, myeloid leukemia of Down syndrome (ML-DS).2 Antecedent MDS is common in those who develop AML in this population, affecting as many as 70% of children with ML-DS.9  
  • Neurofibromatosis type 1 (NF1) is associated with the development of JMML. Patients with NF1 have a 350-fold increased risk of JMML.
  • Shwachman-Diamond syndrome is characterized by pancreatic insufficiency with neutropenia. MDS occurs in 10-25% of individuals with this syndrome.10
  • Fanconi anemia (4-7%) may be a factor;11 48% of patients with Fanconi anemia develop leukemia or MDS by age 40 years. It is often associated with monosomy 7 and duplication of 1q. Diagnosing refractory cytopenia in a patient with Fanconi anemia may be difficult.
  • Familial leukemia (2-6%) may be a factor; JMML is observed in families with monosomy.
  • Kostmann syndrome (0.6%) is congenital agranulocytosis. The survival of patients with this syndrome has significantly improved with the introduction of granulocyte colony-stimulating factor (G-CSF) treatment. Studies from the severe congenital neutropenia registry have shown a 9% crude rate of MDS development and an annual progression rate of 3%.12 Partial or complete loss of chromosome 7 is found in more than half of the patients who develop MDS, and the development of MDS is almost always preceded by acquired mutation of the G-CSF receptor gene.
  • MDS has occasionally been described in patients with Diamond-Blackfan anemia. However, no estimates are available, and it may be rare, given the lack of MDS cases in a study of 229 patients.13
  • Not all bone marrow failure syndromes are associated with the development of MDS (eg, patients with dyskeratosis congenita develop bone marrow failure in 95% of cases, but MDS has only been reported in a few cases).14
  • As a causative factor, previous therapy with alkylating agents (2-5%) is associated with monosomy 7 and chromosome 5 deletions. These patients have poor response rates.
  • Previous administration of a topoisomerase inhibitor is a rare contributing factor. In the rare cases involving a topoisomerase inhibitor, patients usually develop AML.
  • MDS develops in 10-15% of patients with acquired aplastic anemia who are not treated with stem cell transplant; this appears to occur at the same rate in idiopathic and hepatitis-associated aplastic anemia.15 MDS may occur in these cases within 3 years of presentation; whether prolonged treatment with G-CSF and cyclosporine is associated with MDS development is controversial.16

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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