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Query: UMLS:C0026986 (myelodysplastic syndrome)
 14,926 document(s) hit in 31,850,051 MEDLINE articles (0.00 seconds)

Splenomegaly associated with myelodysplastic disorders in children may be massive and can result in pancytopenia, abdominal discomfort, and respiratory distress. When these symptoms cannot be relieved by nonsurgical means, splenectomy may be indicated. Under such conditions, surgical splenectomy carries increased risks, as the thrombocytopenia is difficult to correct secondary to splenic sequestration. Additionally, the surgical anatomy is often distorted secondary to the massive spleen and dissection can be difficult. These factors can lead to uncontrollable hemorrhage. In an attempt to decrease intraoperative blood loss, the authors successfully performed preoperative splenic artery embolization in 11 of 12 children (age range, 1-11 years) with pancytopenia due to hypersplenism. Hypersplenism requiring surgical splenectomy was due to leukemia (n = 9), myelodysplastic syndrome (n = 1), immune thrombocytopenia (n = 1), and osteopetrosis (n = 1). Embolization was performed under general anesthesia, prior to surgery, with gelatin sponge particles alone, Gianturco coils alone, or a combination of polyvinyl alcohol sponge particles and Gianturco coils. Embolization allowed for safe surgical splenectomy.
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PMID:Preoperative embolization of the spleen in children with hypersplenism. 144 26

Three children, two boys and one girl, with Down syndrome (DS) who presented with preleukemia and loss of all or part of chromosome 7 were studied. Initial presentation, with cytopenias and less than 25% blasts in the bone marrow, was between 13 and 30 months of age. Progression to acute nonlymphocytic leukemia occurred 1-8 months after initial presentation. The morphologic type was megakaryoblastic in two, and undifferentiated in one. Two children achieved remission with intensive therapy, and one continues in remission off therapy; the other child died in remission of accidental causes. The third child died of respiratory distress and leukemia after no intervention was chosen. These cases represent the first examples of chromosome 7 abnormalities associated with DS and leukemia, and suggest differences from the "monosomy 7" syndrome seen in children without DS.
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PMID:Chromosome 7 abnormalities in children with Down syndrome and preleukemia. 182 46

We report eight pediatric cases of pulmonary alveolar proteinosis (PAP) that illustrate the polymorphic nature of this disease: two cases with severe neonatal onset, three cases with progressive respiratory distress in patients under 1 year old, and three cases in older children with mild symptoms. Consanguineous parents or affected siblings were identified or suspected in four families. Three patients suffered from associated immune or blood disorders (severe combined immune deficiency, myelodysplasia). The respective roles of a macrophagic dysfunction and of an anomaly of the surfactant are discussed according to the various clinical presentations of pediatric PAP. We performed eight unilateral pulmonary lavages under endoscopy and selective ventilation for two patients under 7 kg in weight. These interventions led to progressive discontinuation of oxygen therapy in one case, and temporarily stabilized the disease for the second. Subsequent recurrence in this second patient was treated by massive lavage under extracorporeal oxygenation. A third infant was successfully transplanted with no recurrence within 3 years. Ambroxol was administered in one case. The three oldest children of our series remained asymptomatic, whereas three of the younger patients died. In the light of this experience, we propose that the treatment administered should be determined according to the age of the patient, the degree of respiratory deficiency, and the nature of any associated pathology.
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PMID:Pulmonary alveolar proteinosis: experience with eight pediatric cases and a review. 915 92

Fifteen patients with a primary myelodysplastic syndrome (MDS) transformed into acute myeloblastic leukemia (AML) were treated with an intensive chemotherapy regimen containing idarubicin. A complete response (CR) was obtained in 10 patients (66.6%). In five of them this was achieved after a single course of chemotherapy. The median time to achieve a CR was 32 days (range 16-42). A partial remission (PR) was obtained in 2 patients after two induction courses of chemotherapy. One patient died during the first induction course following acute respiratory distress syndrome (ARDS) complication, whereas the chemotherapy regimen failed in 2 patients. A short interval between MDS and transformation into AML was associated with a better chance of achieving a CR. Age, karyotype, type of MDS, peripheral blood or bone marrow findings appeared to have no influence on the response to treatment. The median event free survival for patients who achieved CR was 15 months and the median actuarial survival 18 months. These preliminary results need to be confirmed in a multicentre prospective study comparing idarubicin with other anthracyclines.
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PMID:Idarubicin in combination with cytarabine and VP-16 in the treatment of post myelodysplastic syndrome acute myeloblastic leukemia (MDS-AML). 859 Aug 49

Serious hematological diseases often cause respiratory disorders. Because these are related to the prognoses of patients with hematological diseases, their early diagnosis is necessary. This study describes a 6-year-old girl with myelodysplastic syndrome complicated by pulmonary alveolar proteinosis who showed a remarkable increase in her serum KL-6 level. Three years and 2 months after the end of therapy for neonatal melanoma, a diagnosis of myelodysplastic syndrome with leukemic change was made. Ten months after the onset of leukemia, she had respiratory distress with an increased serum KL-6 level of 75,000 U/mL (reference range; < 500 U/mL). Despite various treatments for pulmonary complications, she died 3 months after developing respiratory distress. A diagnosis of pulmonary alveolar proteinosis was made at autopsy. Earlier treatment of respiratory distress could be achieved if serum KL-6 levels were examined earlier.
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PMID:A case of myelodysplastic syndrome complicated by pulmonary alveolar proteinosis with a high serum KL-6 level. 1040 75

In this prospective, multicenter, phase 2 study, multiple myeloma (MM) patients with primary resistant disease or recurrent chemosensitive disease, in chemoresistant relapse, or in second or subsequent remission were treated with high-dose chemoradiotherapy followed by autologous peripheral blood stem cell (PBSC) rescue. PBSCs were collected using granulocyte-macrophage colony-stimulating factor (GM-CSF) 5 microg/kg per day subcutaneously for 3 days. Patients underwent high-dose chemoradiotherapy consisting of melphalan (140 mg/m2 x 1 day), cyclophosphamide (60 mg/kg per day x 2 days), methylprednisolone (2 g/d x 7 days), and total body radiation (150 cGy bid x 3 days) followed by peripheral blood stem cell reinfusion (> or = 1.2 x 10(9) mononucleated cells per kg) and GM-CSF support (5 microg/kg per day) and were evaluated for response, survival, and toxicity. Thirty-six patients, median age 53.4 years, completed the study. The mean pretransplantation cumulative melphalan dose was 464 +/- 72 mg. Excluding the 3 patients (8.3%) who failed to engraft, the median times to engraftment and platelet recovery were 10 days (range, 8-39 days) and 17 days (range, 7-67 days), respectively. Four patients (11.1%) died of complications related to the regimen (main causes of death, sepsis and acute respiratory distress syndrome) within the first 100 days. Twenty-two patients (61.1%) achieved complete response (CR), 8 (22.2%) partial response, and 2 (5.5%) no response. Two patients developed myelodysplastic syndrome after achieving CR. For all 36 patients, the probability of overall survival at 5 years was 27.3%. Median survival was 31 months (range, 0.3-81 months) in all patients and 42 months (range, 3.4-81 months) in those with CR. The probabilities of overall and disease-free survival at 5 years for the 22 patients who achieved CR were 43.6% and 15.7%, respectively. This high-dose chemotherapy regimen coupled with PBSC rescue is associated with a high CR rate and is capable of inducing long-term survival in a subset of heavily pretreated patients with primary resistant or recurrent MM.
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PMID:Treatment of primary resistant or relapsed multiple myeloma with high-dose chemoradiotherapy, hematopoietic stem cell rescue, and granulocyte-macrophage colony-stimulating factor. 1097 14

A 57-year-old man presented with pneumonia, respiratory distress, and myelodysplastic syndrome. A diagnosis of Legionnaires' disease due to Legionella pneumophila (L. pneumophila) was established. The patient had long been drinking tap water via a conduit from a hot spring resource, from which L. pneumophila was also isolated. Both the patient's strain and the water strain of L. pneumophila were identified as serogroup 1, and the genetic relatedness between the two strains as seen by pulsed-field gel electrophoresis was 87%. The patient was successfully treated with erythromycin, fluoroquinolone, and rifampicin. This case raises an important issue on public health represented by legionellosis in Japan.
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PMID:Legionnaires' disease associated with habitual drinking of hot spring water. 1168 36

A 69-year-old man with Sweet's syndrome and myelodysplastic syndrome presented with pneumonia and respiratory distress. He had been taking corticosteroids and methotrexate. The diagnosis of Legionnaires' disease was established by the isolation of Legionella pneumophila serogroup 6 from sputum and a fourfold seroconversion of Legionella antibodies to 1:512. Legionella pneumophila serogroup 6 was isolated from faucets in two homes owned by the patient. Strains of Legionella pneumophila serogroup 6 isolated from the patient's sputum and from one home were demonstrated to be genetically identical by pulsed-field gel electrophoresis but different from strains found in the other home and in a hospital outpatient clinic that he visited. This case illustrates an emerging public health issue concerning acquisition of community-acquired Legionnaires' disease from the homes of immunocompromised hosts. This is the first such case reported in Asia.
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PMID:Residential water supply as a likely cause of community-acquired Legionnaires' disease in an immunocompromised host. 1241 68

Our objective was to study the outcome of allogeneic hematopoietic stem cell transplantation (HSCT) for Shwachman-Diamond Syndrome (SDS). Among 71 SDS patients included in the French Severe Chronic Neutropenia Registry, 10 received HSCT between 1987 and 2004 in five institutions. The indications were bone marrow failure in five cases, and myelodysplastic syndrome (MDS) or leukemia in five cases. The median follow-up of patients who survived without relapse is 6.9 years (3.1-16.8 years). The conditioning regimen consisted of a busulfan-cyclophosphamide combination (n=6) or total body irradiation plus chemotherapy (n=4). Six patients received stem cells from unrelated donors and four from identical siblings. Engraftment was complete in eight patients and unassessable in two patients. These latter two patients died of infections 32 and 36 days after HSCT, with grade IV graft-versus-host disease and multiorgan dysfunction. A third patient died from an acute respiratory distress syndrome 17 months after HSCT with progressive granulocytic sarcoma. One patient had an MDS relapse 4 months after HSCT and died 10 months later. The overall 5-year event-free survival rate is 60+/-15%. We conclude that HSCT is feasible for patients with SDS who develop bone marrow failure or malignant transformation.
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PMID:Hematopoietic stem cell transplantation for Shwachman-Diamond syndrome: experience of the French neutropenia registry. 1615 25

The DNA hypomethylating agent azacitidine was approved by the United States Food and Drug Administration after the drug demonstrated superiority over the best supportive care for treatment of myelodysplastic syndrome in patients unable to undergo stem cell transplantation. Mild adverse reactions, both hematologic and nonhematologic, are not uncommon; however, severe adverse effects are rare. We describe a 55-year-old woman who was treated with azacitidine for myelodysplastic syndrome and experienced hyperthermia that was not attributable to other causes. The patient's treatment course was further complicated by interstitial pneumonitis and hypoxic respiratory failure that ultimately led to acute respiratory distress syndrome. Hyperthermia develops when discord occurs between metabolic heat production and heat dissipation. The process of temperature regulation can be altered by drugs such as succinylcholine, phenothiazines, monoamine oxidase inhibitors, atropine, benztropine, antihistamines, cocaine, Ecstasy, amphetamines, and haloperidol. The hyperthermia in this patient was refractory to antipyretic therapy and was not due to other drug-induced hyperthermic syndromes. She eventually responded to high-dose methylprednisolone. The Naranjo adverse drug reaction probability scale score indicated that the association between azacitidine and hyperthermia was probable. Clinicians should be aware of this rare, severe, potential adverse effect of azacitidine.
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PMID:Azacitidine-associated hyperthermia and interstitial pneumonitis in a patient with myelodysplastic syndrome. 1804 93


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