The myelodysplastic syndromes (MDS) are characterized by ineffective hematopoiesis associated with multilineage cytopenias leading to serious morbidity or mortality, and the additional risk of leukemic transformation. The management of patients with MDS can be very complex and varies according to both the clinical manifestations in individual patients as well as the presence of complicating medical conditions. However, therapeutic dilemmas still exist for MDS due to the multifactorial pathogenetic features of the disease, its heterogeneous stages, and the elderly patient population. For these reasons, proper guidelines for management are necessary. This review describes the proper diagnosis for MDS, decision-making approaches for optimal therapeutic options that are based on a consideration of patient clinical factors and risk-based prognostic categories, and the use of recently available biospecific drugs such as hypomethylating agents that are potentially capable of abrogating the abnormalities associated with MDS. Proper indications and methods for transplantation, response criteria, management for iron overload for highly transfused patients and specific considerations for MDS in childhood are also described. All of these topics were discussed at the third symposium of AML/MDS working party on 3 March, 2007.
Aged ; Diagnosis ; Hematopoiesis ; Humans ; Iron Overload ; Mortality ; Myelodysplastic Syndromes* ; Transplantation

No abstract available.
Chromosome Aberrations ; Humans ; Karyotype ; Leukemia, Myeloid, Acute/*diagnosis/etiology/mortality ; Myelodysplastic Syndromes/complications/*diagnosis ; Myeloproliferative Disorders/complications/*diagnosis ; Philadelphia Chromosome ; Survival Rate

Myelodysplastic syndromes (MDS) are probably the most common hematologic malignancies in adults over the age of 60 and are a major source of morbidity and mortality among older age groups. Diagnosis and management of this chronic blood cancer has evolved significantly in recent years and there are Food and Drug Administration-approved therapies that can extend patients' life expectancy and improve quality of life. Primary care physicians (PCPs) are often involved in the process of diagnosis and follow-up of MDS patients, especially those in low-risk groups. They can therefore play an important role in improving patient care and quality of life by ensuring early referral and participating in supportive management. There is also a shortage of oncologists which increases the importance of the role of PCPs in management of MDS patients. In the face of limited resources, PCPs can improve access and quality of care in MDS patients. This article provides an overview of the common manifestations, diagnostic approaches, and therapeutic modalities of MDS for PCPs, with a focus on when to suspect MDS, when a referral is appropriate, and how to provide appropriate supportive care for patients diagnosed with MDS.
Adult ; Bone Marrow Diseases ; Diagnosis ; Follow-Up Studies ; Hematologic Neoplasms ; Humans ; Life Expectancy ; Mortality ; Myelodysplastic Syndromes* ; Patient Care ; Physicians, Primary Care ; Primary Health Care* ; Quality of Life ; Referral and Consultation

BACKGROUND: The presence of significant dysplasia in bone marrow (BM) aspirates helps to distinguish between hypocellular myelodysplastic syndrome (hMDS) and aplastic anemia (AA). Occasionally, diluted BM aspirates make it difficult to recognize dysplastic changes and can also negatively affect the detection of cytogenetic abnormalities in hMDS. We evaluated the usefulness of CD34 and p53 immunoreactivity for discriminating between hMDS and AA and for estimating survival outcomes in hMDS patients. METHODS: BM clot section (BMC) or BM biopsy (BMB) specimens were obtained from 64 hMDS/AA patients (33 with hMDS and 31 with AA) and seven controls. Immunohistochemical (IHC) staining for CD34 and p53 was performed by using the EnVision detection system (Dako, Denmark). We compared the results of IHC staining, BM findings, and chromosomal analyses, and determined overall survival outcomes. RESULTS: The number of CD34- and p53-positive BM cells was higher among the patients with hMDS than among the patients with AA (P<0.001 and P=0.001, respectively). hMDS patients with increased CD34-positive cells had significantly poorer survival outcomes compared with those with normal number of CD34-positive cells (P=0.013). CONCLUSIONS: CD34 and p53 IHC stains of BMC or BMB provide useful information for differentiating between hMDS and AA. CD34 IHC staining of BMC or BMB also provides useful information for estimating survival outcomes in hMDS patients.
Adolescent ; Adult ; Anemia, Aplastic/*diagnosis ; Antigens, CD34/*metabolism ; Bone Marrow/metabolism/*pathology ; Child ; Chromosome Aberrations ; Diagnosis, Differential ; Female ; Humans ; Immunohistochemistry ; Kaplan-Meier Estimate ; Male ; Middle Aged ; Myelodysplastic Syndromes/*diagnosis/mortality ; ROC Curve ; Tumor Suppressor Protein p53/*metabolism

PURPOSE: Hepatic veno-occlusive disease (VOD) is a life-threatening complication occurring early after stem cell transplantation (SCT). Early diagnosis and effective treatment has not been established in severe VOD. Because there are few reports on VOD in Korean children, we evaluated the clinical characteristics of VOD following SCT in children. METHODS: We retrospectively reviewed the chart of all patients (n=116) receiving SCTs in CNUH Pediatric BMT center between May, 1991 and June, 2004. RESULTS: VOD developed in 11 patients (9.5%) (median age, 9.8 years; range, 2 to 13.9). Underlying diagnoses were ALL (n=3), severe aplastic anemia (n=3), AML (n=2), acute biphenotypic leukemia (n=1), neuroblastoma (n=1), and myelodysplastic syndrome (n=1). The median day of onset of VOD was D+9 (range, D-3 to D+19). VOD was classified as moderate in 5 and severe in 6 cases. Maximum level of serum total bilirubin was 2.9 mg/dL (range, 2.1 to 9.2) in moderate VOD and 7.3 mg/dL in severe VOD (range, 2.0 to 24.2) at D+18 (range, D-5 to D+59). We successfully treated VOD with various combinations including tPA and heparin (2/5, 40%), ursodeoxycholic acid (2/5, 40%), N-acetylcysteine (3/5, 60%), and defibrotide (1/2, 50%). All of 5 patients with moderate VOD survived at D+100 (range, 5.5+ to 66.6+ months). Five of 6 (83%) patients with severe VOD died within first 19 day from complications of VOD. CONCLUSION: This retrospective study showed that the incidence of VOD was 9.5%, and the mortality of severe VOD was still high which would necessitate early diagnosis, effective prevention and treatment.
Acetylcysteine ; Anemia, Aplastic ; Bilirubin ; Child ; Diagnosis ; Early Diagnosis ; Hematopoietic Stem Cell Transplantation* ; Hematopoietic Stem Cells* ; Heparin ; Hepatic Veno-Occlusive Disease* ; Humans ; Incidence ; Leukemia, Biphenotypic, Acute ; Mortality ; Myelodysplastic Syndromes ; Neuroblastoma ; Retrospective Studies* ; Stem Cell Transplantation ; Ursodeoxycholic Acid

BACKGROUND: AML with myelodysplasia related changes (AML MRC) is known to show a poor prognosis compared with de novo AML, but controversies exist about the prognostic impact of multilineage dysplasia (MLD) among MRC. We investigated the prognostic impact of MLD in AML MRC. METHODS: A total of 357 patients newly diagnosed as AML at Asan Medical Center from January 2001 to December 2005 were analyzed. They were diagnosed and classified as AML with recurrent genetic abnormalities, AML MRC, and AML not otherwise specified (AML NOS). Prognostic markers including overall survival (OS) and event free survival (EFS) were obtained through retrospective analysis of electronic medical records. RESULTS: AML MRC patients showed a lower complete remission (CR) rate (44.7% vs. 64.9%, P=0.002) and shorter OS (297 vs. 561 days, P=0.004) and EFS (229 vs. 374 days, P=0.004) than AML NOS patients. Patients with MLD among AML MRC also showed a lower CR rate (37.7%, P=0.001) and shorter OS (351 days, P=0.036) and EFS (242 days, P=0.076) than AML NOS patients. However, among AML MRC patients, there were no differences in OS, EFS and CR between patients with and without MLD. CONCLUSIONS: AML MRC patients showed a lower CR rate and shorter OS and EFS than AML NOS patients. AML MRC patients with MLD showed similar results and their prognosis was not different from those without MLD. MLD findings among AML MRC could be an independent poor prognostic factor in de novo AML.
Adolescent ; Adult ; Aged ; Aged, 80 and over ; Cell Lineage ; Child ; Child, Preschool ; Data Interpretation, Statistical ; Disease-Free Survival ; Female ; Humans ; Infant ; Leukemia, Myeloid, Acute/complications/diagnosis/*mortality ; Male ; Middle Aged ; Myelodysplastic Syndromes/complications/*diagnosis ; Prognosis ; Retrospective Studies ; Survival Analysis

This retrospective study investigated the clinical characteristics and outcomes of second malignant neoplasms (SMNs) in survivors of childhood cancer from multiple institutions in Korea. A total of 102 patients from 11 institutions who developed SMN after childhood cancer treatment between 1998 and 2011 were retrospectively enrolled. The most common primary malignant neoplasms (PMNs) were central nervous system (CNS) tumors (n = 17), followed by acute lymphoblastic leukemia (n = 16), non-Hodgkin lymphoma (n = 13), and osteosarcoma (n = 12). The most common SMNs were therapy-related myeloid neoplasms (t-MNs; acute myeloid leukemia [AML], 29 cases; myelodysplastic syndrome [MDS], 12 cases), followed by thyroid carcinomas (n = 15) and CNS tumors (n = 10). The median latency period was 4.9 years (range, 0.5-18.5 years). Among 45 patients with solid tumors defined as an SMN, 15 (33%) developed the lesion in a field previously subjected to radiation. The 5-year overall survival (OS) rate of patients with an SMN was 45% with a median follow-up time of 8.6 years. Patients with AML, MDS, and CNS tumors exhibited the poorest outcomes with 5-year OS rates of 18%, 33%, and 32%, respectively, whereas those with second osteosarcoma showed comparable outcomes (64%) to patients with primary counterpart and those with second thyroid carcinoma had a 100% OS rate. Further therapeutic efforts are recommended to improve the survival outcomes in patients with SMNs, especially in cases with t-MNs and CNS tumors.
Adolescent ; Antineoplastic Agents/therapeutic use ; Central Nervous System Neoplasms/diagnosis/drug therapy/radiotherapy ; Child ; Child, Preschool ; Disease-Free Survival ; Hospitals ; Humans ; Infant ; Leukemia, Myeloid, Acute/diagnosis/epidemiology/mortality/therapy ; Myelodysplastic Syndromes/diagnosis/epidemiology/mortality/therapy ; Neoplasms, Second Primary/*diagnosis/epidemiology/mortality/therapy ; Osteosarcoma/diagnosis/epidemiology ; Retrospective Studies ; Stem Cell Transplantation ; Survival Rate ; Transplantation, Autologous ; Young Adult

This retrospective study investigated the clinical characteristics and outcomes of second malignant neoplasms (SMNs) in survivors of childhood cancer from multiple institutions in Korea. A total of 102 patients from 11 institutions who developed SMN after childhood cancer treatment between 1998 and 2011 were retrospectively enrolled. The most common primary malignant neoplasms (PMNs) were central nervous system (CNS) tumors (n = 17), followed by acute lymphoblastic leukemia (n = 16), non-Hodgkin lymphoma (n = 13), and osteosarcoma (n = 12). The most common SMNs were therapy-related myeloid neoplasms (t-MNs; acute myeloid leukemia [AML], 29 cases; myelodysplastic syndrome [MDS], 12 cases), followed by thyroid carcinomas (n = 15) and CNS tumors (n = 10). The median latency period was 4.9 years (range, 0.5-18.5 years). Among 45 patients with solid tumors defined as an SMN, 15 (33%) developed the lesion in a field previously subjected to radiation. The 5-year overall survival (OS) rate of patients with an SMN was 45% with a median follow-up time of 8.6 years. Patients with AML, MDS, and CNS tumors exhibited the poorest outcomes with 5-year OS rates of 18%, 33%, and 32%, respectively, whereas those with second osteosarcoma showed comparable outcomes (64%) to patients with primary counterpart and those with second thyroid carcinoma had a 100% OS rate. Further therapeutic efforts are recommended to improve the survival outcomes in patients with SMNs, especially in cases with t-MNs and CNS tumors.
Adolescent ; Antineoplastic Agents/therapeutic use ; Central Nervous System Neoplasms/diagnosis/drug therapy/radiotherapy ; Child ; Child, Preschool ; Disease-Free Survival ; Hospitals ; Humans ; Infant ; Leukemia, Myeloid, Acute/diagnosis/epidemiology/mortality/therapy ; Myelodysplastic Syndromes/diagnosis/epidemiology/mortality/therapy ; Neoplasms, Second Primary/*diagnosis/epidemiology/mortality/therapy ; Osteosarcoma/diagnosis/epidemiology ; Retrospective Studies ; Stem Cell Transplantation ; Survival Rate ; Transplantation, Autologous ; Young Adult

BACKGROUND: Mucormycosis is a highly virulent and rapidly progressive infectious disease caused by Mucorales. Immunocompromised hosts, such as patients with poorly controlled diabetes mellitus or diabetic ketoacidosis, patients receiving long-term deferoxamine therapy, and patients with hematologic malignancy, are predisposed to mucormycosis. We presented a case of brain abscess in a patient with myelodysplastic syndrome, and reviewed the cases of mucormycosis reported in Korea. METHODS: Relevant reports on mucormycosis were collected by searching the Korean database of medical literature. A total of 57 cases from 41 reports in Korea were reviewed as to clinical types, predisposing factors, treatments, and outcomes. RESULTS: The male to female ratio was 1.2:1. The mean age was 44 (range 1-72) years. The most frequent predisposing factor was diabetes mellitus (40 %), followed by the use of immunosuppressive agents (21%), and hematologic malignancies (16%). The most frequent clinical type was rhinocerebral (65%), followed by gastrointestinal (12%), pulmonary (9%), cutaneous (7%), and disseminated type (5%). The overall mortality rate was 33.3%, and the mortality rate in patients treated with surgical debridement was lower that in patients treated medically. The mortality rate in patients receiving surgical debridement only 13.3%, surgical debridement plus amphotericin B 26.9%, amphotericin B only 44.4%, and supportive care only 85.7%. Patients with disseminated type had a higher mortality rate than the other types. Conclusions:Early diagnosis of mucormycosis followed by the removal of predisposing factors and aggressive management, such as early surgical debridement and use of amphotericin B greatly affect therapeutic outcome. Therefore, much attention to the clinical features and identification of the organism are warranted. Collaborative evaluation through the collection of more cases with mucormycosis may be required in order to clarify the characteristics of mucormycosis in Korea.
Amphotericin B ; Brain Abscess ; Causality ; Communicable Diseases ; Debridement ; Deferoxamine ; Diabetes Mellitus ; Diabetic Ketoacidosis ; Diagnosis ; Female ; Hematologic Neoplasms ; Humans ; Immunocompromised Host ; Immunosuppressive Agents ; Korea* ; Male ; Mortality ; Mucorales ; Mucormycosis* ; Myelodysplastic Syndromes*

BACKGROUND: Mucormycosis is a highly virulent and rapidly progressive infectious disease caused by Mucorales. Immunocompromised hosts, such as patients with poorly controlled diabetes mellitus or diabetic ketoacidosis, patients receiving long-term deferoxamine therapy, and patients with hematologic malignancy, are predisposed to mucormycosis. We presented a case of brain abscess in a patient with myelodysplastic syndrome, and reviewed the cases of mucormycosis reported in Korea. METHODS: Relevant reports on mucormycosis were collected by searching the Korean database of medical literature. A total of 57 cases from 41 reports in Korea were reviewed as to clinical types, predisposing factors, treatments, and outcomes. RESULTS: The male to female ratio was 1.2:1. The mean age was 44 (range 1-72) years. The most frequent predisposing factor was diabetes mellitus (40 %), followed by the use of immunosuppressive agents (21%), and hematologic malignancies (16%). The most frequent clinical type was rhinocerebral (65%), followed by gastrointestinal (12%), pulmonary (9%), cutaneous (7%), and disseminated type (5%). The overall mortality rate was 33.3%, and the mortality rate in patients treated with surgical debridement was lower that in patients treated medically. The mortality rate in patients receiving surgical debridement only 13.3%, surgical debridement plus amphotericin B 26.9%, amphotericin B only 44.4%, and supportive care only 85.7%. Patients with disseminated type had a higher mortality rate than the other types. Conclusions:Early diagnosis of mucormycosis followed by the removal of predisposing factors and aggressive management, such as early surgical debridement and use of amphotericin B greatly affect therapeutic outcome. Therefore, much attention to the clinical features and identification of the organism are warranted. Collaborative evaluation through the collection of more cases with mucormycosis may be required in order to clarify the characteristics of mucormycosis in Korea.
Amphotericin B ; Brain Abscess ; Causality ; Communicable Diseases ; Debridement ; Deferoxamine ; Diabetes Mellitus ; Diabetic Ketoacidosis ; Diagnosis ; Female ; Hematologic Neoplasms ; Humans ; Immunocompromised Host ; Immunosuppressive Agents ; Korea* ; Male ; Mortality ; Mucorales ; Mucormycosis* ; Myelodysplastic Syndromes*