OBJECTIVE: To investigate the efficacy and safely of DAC and CAG/HAG preexcitation chemotherapy regimens for the treatment of patients with MDS-RAEB (refractory anemia with excess blasts, RAEB). METHODS: The clinical data of 86 MDS-RAEB patients were analyzed retrospectively from February 2014 to February 2018. According to therapeutic regimem, the 86 patients were divided into 2 groups: group A (41 patients) with DAC preexcitation chemotherapy regimen, and group B (45 patients) with CAG/HAG preexcitation chemotherapy regimen; and the disease control effect, effective treatment course, median survival time and incidence of adverse reactions were compared between these 2 groups. RESULTS: The CR rate and ORR rate were not significantly different between these 2 groups (P＞0.05). The mCR rate in group A was significantly higher than that in group B (P＜0.05). The numbers of cases obtained therapeutic efficacy at 2 rd and 3 rd conrse in group A significantly more than those in group B (P＜0.05), but the number of cases obtained efficacy at 1 st course in group B was significantly higher than that in group A (P＜0.05). The median OS time was not significanly different between 2 groups (P＞0.05). The duration of neutrophils deficiency in group A was significantly shorter than that in group B (P＜0.05). The transfusion volume of red blood cells and platelets in group A was significantly less than that of group B (P＜0.05). The incidence of neutropenia, anemia and thrombocytopenia of III-IV grade at different treatment courses of group A were significantly lower than that in group B (P＜0.05). The incidence of infection of III-IV grade in group A at 3rd treatment course was significantly lower than that in group B (P＜0.05). CONCLUSION Preexcitation chemotherapy regimens of DAC and CAG/HAG for the treatment of MDS-RAEB possess the same effects for disease control; application of DAC regimen can efficiently reduce the risk of adverse reaction, but CAG/HAG regimen can be helpful to accelerate the effective process of treatment.
Anemia, Refractory ; Anemia, Refractory, with Excess of Blasts ; drug therapy ; Humans ; Myelodysplastic Syndromes ; drug therapy ; Retrospective Studies ; Treatment Outcome

Refractory anemia with ringed sideroblasts (RARS) is an extremely rare type of myelodysplastic syndrome in children. We describe a 10-year-old boy with RARS presented with pancytopenia. He remained relatively stable with only a few transfusions until age of 20 years, when he underwent an allogeneic bone marrow transplantation (BMT) because of increased transfusion requirements. He remains in complete chimeric state at 20 months posttransplant with normal hematologic parameters. To our knowledge, this is the first description of successful BMT in a patient with childhood-onset RARS. The indication of BMT for this rare disorder in children is discussed.
Anemia, Refractory/therapy* ; Anemia, Sideroblastic/therapy* ; Bone Marrow Transplantation* ; Case Report ; Child ; Human ; Male ; Transplantation, Homologous

OBJECTIVETo explore the efficiency and side-effects of the combination of cyclosporine A (CsA) and thalidomide in patients with myelodysplastic syndromes (MDS).

METHODSA total of thirty-seven patients with MDS-RCMD or-RAEB-I were treated with CsA in combination with thalidomide. The initial CsA dose of 3 mg×kg(-1)×d(-1) was administered, all patients had their CsA blood concentration concurrently monitored until it reached and maintained between 100 and 200 µg/L. The initial dose of thalidomide was 50 mg/d, with increasing dose of 50 mg every week until the maximum of 200 mg/d. The hematological response was assessed according to the modified criteria of the International Working Group, and adverse events were graded with the Common Toxicity Criteria (v3.0) of the National Cancer Institute. The response duration and overall survival of the patients were also observed.

RESULTS19/37 cases (51.4%) achieved hematologic improvement (HI)-erythroid response (HI-E), 9/29 cases (31.0%) HI-platelet response (HI-P) and 7/33 cases (21.2%) HI-neutrophil response (HI-N). 15 of 32 transfusion-dependent patients (46.9%) achieved transfusion independence. The median response duration of HI-E, HI-P and HI-N were 88 (4 - 88) weeks, 78 (8 - 84(+)) weeks and 78 (10 - 84(+)) weeks respectively. The median overall survival was 52 months on a 29 (4 - 103) months median follow-up. Some patients developed grades I-II hepatic or nephritic impairment, constipation, lethargy, dizziness, edema, rashes or numbness, and all were tolerable and reversible. No grade III or severer adverse events were observed.

CONCLUSIONCsA in combination with thalidomide appears to be effective mainly in inducing HI-E and relatively well-tolerated for the treatment of patients with MDS.

Anemia, Refractory, with Excess of Blasts ; drug therapy ; Cyclosporine ; therapeutic use ; Humans ; Myelodysplastic Syndromes ; drug therapy ; Thalidomide ; therapeutic use ; Treatment Outcome

In thirty patients with acute leukemia and severe aplastic anemia receiving random single donor platelet transfusions, the development of refractoriness by consecutive platelet transfusions with cytapheresis and its relationship to the appearance of anti-platelet antibodies were investigated. The median number of platelet transfusions inducing refractoriness was 13 times, and 20% of the patients remained unrefractory despite of the repeated multiple platelet transfusions up to 20 to 25 times. The results of anti-platelet antibody tasts by the enzyme-linked immunosorbent assay(ELISA) and immunofluorescent techniques(IFT) showed no statistically significant relationship with the refractoriness (p greater than 0.1). Although there was significant correlation between the results of ELISA and IFT, both tests were insufficient to find out refractoriness even with the use of pooled platelets from multiple donors as target cells. This study shows that 13 single donor platelet transfusions result in refractoriness, that both ELISA and IFT are insufficient to detect refractoriness despite of their significant correlation, and that other methods than these are needed in order to detect alloimmunization.
Adolescent ; Adult ; Aged ; Anemia, Aplastic/therapy ; Anemia, Refractory/*etiology ; Antibodies/metabolism ; *Blood Platelets/immunology ; *Blood Transfusion ; Female ; Humans ; Leukemia/therapy ; Male ; Middle Aged

Anemia, Refractory, with Excess of Blasts ; complications ; drug therapy ; Azacitidine ; analogs & derivatives ; therapeutic use ; Humans ; Male ; Middle Aged ; Myelodysplastic Syndromes ; complications ; drug therapy ; Treatment Outcome

To explore the treatment of refractory anemia (RA), 7 cases of myelodysplastic syndrome (subtype of refractory anemia) were treated in combination of cyclosporin A (CsA) with stanozolol. Duration of treatment with CsA was 5 months-3 years (mean 13 months). The results showed that among 7 cases 6 were effective, 1 case no responded to treatment. 3 cases out of 6 effective cases achieved complete remission without transfusion dependence, 1 cases achieved partial remission, 2 cases were improved. During the investigation signs of leukemia ot other malignant tumors not were found in all cases. In conclusion, CsA treatment is effective for part cases of RA, side effects of drugs are tolerable for patients.
Adolescent ; Adult ; Aged ; Anemia, Refractory ; drug therapy ; Cyclosporine ; administration & dosage ; Drug Therapy, Combination ; Female ; Humans ; Male ; Middle Aged ; Stanozolol ; administration & dosage

Acute Disease ; Anemia, Refractory ; etiology ; therapy ; Child ; Child, Preschool ; Cord Blood Stem Cell Transplantation ; methods ; Female ; Histocompatibility Testing ; Humans ; Leukemia ; complications ; pathology ; therapy ; Male ; Treatment Outcome

Invasive pulmonary aspergillosis is difficult to diagnose and a critical ill with high mortality. In this paper, the diagnosis and treatment of invasive pulmonary aspergillosis complicated in 3 cases of hematological malignancy (2 acute leukemias and 1 MDS-RA) were retrospectively analysed. All patients had histories of hypoimmunity and were received prophylactic antifungal treatment. Pulmonary aspergillosis infection still occurred and confirmedly diagnosed by sputum examination. After 7 to 14 days of combination treatment of liposomal amphotericin B, itraconazole and flucytosine, 2 cases were cured and another showed effective. In conclusion, early diagnosis and treatment of invasive pulmonary aspergillosis are very critical and the therapeutic effectiveness of combined scheme with liposomal amphotericin B, itraconazole and flucytosine is very effective for pulmonary aspergillosis.
Adult ; Amphotericin B ; therapeutic use ; Anemia, Refractory ; complications ; Aspergillosis ; diagnosis ; drug therapy ; Female ; Humans ; Leukemia ; complications ; Lung Diseases, Fungal ; diagnosis ; drug therapy ; Male ; Middle Aged

Here, we report on a 20-year-old patient with a primary nonseminomatous mediastinal germ cell tumor (MGCT) who developed myelodysplastic syndrome (MDS) 2 months following chemotherapy with cisplatin, etoposide, ifosfamide, and paclitaxel. Bone marrow examinations revealed that the MDS was a refractory anemia with excess type II blasts and complex chromosomal abnormalities. With the onset of MDS occurring rapidly following chemotherapy, it is unlikely to have been caused by the therapy. We discuss the association between primary nonseminomatous MGCTs and hematological malignancies, including the possibility of a common clonal origin.
Anemia, Refractory ; Bone Marrow Examination ; Chromosome Aberrations ; Cisplatin ; Drug Therapy* ; Etoposide ; Germ Cells* ; Hematologic Neoplasms ; Humans ; Ifosfamide ; Myelodysplastic Syndromes* ; Neoplasms, Germ Cell and Embryonal* ; Paclitaxel ; Young Adult

To determine the efficacy and tolerance to cyclosporine A (CsA) based therapy in patients with myelodysplastic syndrome (MDS), 16 patients with MDS consisting of 10 refractory anemia (RA) and 6 refractory anemia with accessory blasts less than 10% (RAEB-1) were analyzed. Five patients had hypocellular bone marrows and 11 patients had normocellular or hypercellular marrows. The dose of CsA was 2.5-5.5 mg/(kg.d) for 2 weeks to 2 years (mean 8 months). Two out of 16 patients were treated with CsA alone, 14 patients were treated with CsA, recombinant human erythropoietin, androgens, 1, 25 dihydroxy vitamin D(3) or two or three of them combination with CsA. Treatment responses were classified according to the International Working Group (IWG) criteria as complete remission (CR), partial remission (PR), hematological improvement (HI) and no response (NR). Patients who obtained CR, PR or HI were defined as responders. The results showed that HI was observed in 12 patients, PR in 2 patients and NR in 2 patients. Total response rate was 87.5%. Response rates shown in neutrophil lineage, platelet and erythroid lineage were 83.3%, 66.7% and 60%, respectively; their shortest time required to obtain some hematologic improvement after initiation of CsA therapy was 2 weeks, 1 month and 1 month, respectively. Of 13 patients being transfusion-dependent before treatment, 3 patients did not need transfusion any more and 5 showed the reduced transfusion requirements after CsA therapy. In 10 patients with RA, 9 responded to CsA. Of 6 patients with RAEB, 1 patient had no response and died of RAEB-t and 5 patients had transient responses. One of the latter transformed to CMML and two relapsed. The total response rate decreased to 50% in the patients with CsA therapy lasting more than 3 months at the end of following-up. The adverse effects included hirsutism, hyperplastic gingiva, reversible hepatic and renal dysfunction. In conclusion, the usefulness of CsA based therapy for MDS-RA and RAEB-1 with any marrow cellularity is useful, the CsA dose of 3-5 mg/(kg.d) is safe and efficacious.
Adolescent ; Adult ; Aged ; Androgens ; administration & dosage ; Anemia, Refractory ; drug therapy ; Anemia, Refractory, with Excess of Blasts ; drug therapy ; Calcitriol ; administration & dosage ; therapeutic use ; Cyclosporine ; administration & dosage ; therapeutic use ; Drug Therapy, Combination ; Erythropoietin ; administration & dosage ; therapeutic use ; Female ; Humans ; Immunosuppressive Agents ; administration & dosage ; therapeutic use ; Male ; Middle Aged ; Myelodysplastic Syndromes ; drug therapy ; Recombinant Proteins ; Treatment Outcome