OBJECTIVES: To study the association between paroxysmal nocturnal hemoglobinuria (PNH) clone and immunosuppressive therapy (IST) in children with severe aplastic anemia (SAA). METHODS: A retrospective analysis was performed on the medical data of 151 children with SAA who were admitted and received IST from January 2012 to May 2020. According to the status of PNH clone, these children were divided into a negative PNH clone group (n=135) and a positive PNH clone group (n=16). Propensity score matching was used to balance the confounding factors, and the impact of PNH clone on the therapeutic effect of IST was analyzed. RESULTS: The children with positive PNH clone accounted for 10.6% (16/151), and the median granulocyte clone size was 1.8%. The children with positive PNH clone had an older age and a higher reticulocyte count at diagnosis (P<0.05). After propensity score matching, there were no significant differences in baseline features between the negative PNH clone and positive PNH clone groups (P>0.05). The positive PNH clone group had a significantly lower overall response rate than the negative PNH clone group at 6, 12, and 24 months after IST (P<0.05). The evolution of PNH clone was heterogeneous after IST, and the children with PNH clone showed an increase in the 3-year cumulative incidence rate of aplastic anemia-PNH syndrome (P<0.05). CONCLUSIONS SAA children with positive PNH clone at diagnosis tend to have poor response to IST and are more likely to develop aplastic anemia-PNH syndrome.
Anemia, Aplastic/drug therapy* ; Child ; Clone Cells ; Hemoglobinuria, Paroxysmal/etiology* ; Humans ; Immunosuppression Therapy ; Retrospective Studies

On patients with disorder of hematopoiesis such as leukemia, aplastic anemia, MDS(Myelodisplastic Syndromes), removal of infectious foci prior to the BMT(Bone Marrow Transplantation) is a necessity and what is more, there is no sufficient time to control the infections because the chemotherapy for BMT should be started as soon as possible. And the transfusion should be minimized to prevent the alloimmunization. In those reasons, oral & maxillofacial surgeons are often in need of multiple extractions, and should take into consideration the possibility of complications after multiples extractions such as infection, severe bleeding because those situations can be fatal on patients with disorder of hematopoiesis. We present our experience in multiple extractions on 30 patients with disorder of hematopoiesis referred from Catholic Hematopoiesis Stem Cell transplantation Center at St. Mary's Hospital, The Catholic University of Korea.
Anemia, Aplastic ; Bone Marrow ; Drug Therapy ; Hematopoiesis* ; Hemorrhage ; Humans ; Korea ; Leukemia ; Stem Cell Transplantation

Background: Aplastic anemia following chemotherapy of acute leukemia is a common complication, which may lead to severe consequences. Objective: To study characteristics of aplastic anemia occurred in ccute myelogenous leukemia (AML) patients, following chemotherapy. Subjects and methods: A prospective study was carried out in 50 AML patients treated at National Institute of Hematology and Blood Transfusion from Aug 2005 to Dec 2006. These patients were treated by induction chemotherapy with "3+7" regime. Result: Aplastic anemia had been seen in 100% patients. Characteristics of this condition were poor marrow cells (average marrow cell count was 15.1\xb112.6 G/l) and strongly decreased counts of hemoglobin, white blood cells and platelets. Hemoglobin, white blood cell and platelet counts at the lowest level were 83.32 g/l; 0.96 G/l; 30.18 G/l; respectively. This situation prolonged for 3-4 weeks and changed into the most severe condition at the end of second week after chemotherapy. Infection frequency was 92%. Conclusion: Aplastic anemia following chemotherapy of AML patients is a common complication with severe consequences such as significant decrease of WBC and platelet counts, which may lead to opportunistic infection. Hence, this complication must be monitored, detected and treated promptly. \r\n', u'\r\n', u'
Leukemia ; Myeloid ; Acute/ pathology ; prevention & ; control ; complications ; drug therapy ; Anemia ; Aplastic/ blood ; complications ; pathology

OBJECTIVE: To evaluate the efficacy and safety of Pai-Neng-Da Capsule (, panaxadiol saponins component, PNDC) in combination with the cyclosporine and androgen for patients with chronic aplastic anemia (CAA). METHODS: A total of 79 CAA patients was randomly divided into 2 groups by a random number table, including PCA group [43 cases, orally PNDC 320 mg/d plus cyclosporine 5 mg/(kg·d) plus andriol 80 mg/d] and CA group [36 cases, orally cyclosporine 5 mg/(kg·d) plus andriol 160 mg/d]. All patients were treated and followed-up for 6 treatment courses over 24 weeks. The complete blood counts, score of Chinese medical (CM) symptoms were assessed and urine routine, electrocardiogram, hepatic and renal function were observed for safety evaluation. Female masculinization rating scale was established according to the actual clinical manifestations to evaluate the accurate degree of masculinization in female CAA patients treated by andriol. RESULTS: The effective rates were 88.1% (37/42) in the PCA group and 77.8% (28/36) in the CA group based on the standard for the therapeutic efficacy evaluation of hematopathy. There was no significant difference in the white blood cell (WBC) counts, platelet counts and hemoglobin concentration of peripheral blood between two groups after 6 months treatment. The masculinization score of female patient in the PCA group was significantly lower than the CA group (P<0.05). The mild abdominal distention was observed in 1 cases in the PCA group. In CA group, the abnormalities in the hepatic function developed in 2 cases and the renal disfunction was found in 1 case. CONCLUSION The PNDC possesses certain curative effects in the treatment of CAA without obvious side-effects and can partially replace andriol thereby to reduce the degree of masculinization [Registried at Chinese Clinical Trial Registry (ChicTR1900028153)].
Androgens ; Anemia, Aplastic/drug therapy* ; China ; Female ; Humans ; Nonprescription Drugs ; Saponins/therapeutic use*

Anemia, Aplastic/drug therapy* ; Animals ; Antilymphocyte Serum ; Child ; Cyclosporine ; Humans ; Immunosuppressive Agents ; Swine ; Treatment Outcome

Anemia, Aplastic ; drug therapy ; etiology ; Humans ; Immunosuppressive Agents ; therapeutic use ; Medicine, Chinese Traditional ; Myelodysplastic Syndromes ; drug therapy ; etiology

Humans ; Cyclosporine/therapeutic use* ; Anemia, Aplastic/drug therapy* ; Neoplasms/drug therapy* ; Immunosuppressive Agents/therapeutic use* ; Benzoates/therapeutic use*

OBJECTIVETo testify whether absolute neutrophil count (ANC) response to preimmunosuppressive-therapy (pre-IST) granulocyte-stimulating factor (G-CSF) treatment could predict early response to IST in severe aplastic anemia (SAA).

METHODSClinical data and hematologic response of 125 SAA patients treated with antithymocyte globulin (r-ATG) combined with cyclosporine were retrospectively analyzed. Correlation of ANC response to pre-IST G-CSF treatment and early response to IST were statistically analyzed, and receiver operating characteristic (ROC) curve was used to estimate the value of increased ANC (∆ANC) in predicting early IST response.

RESULTSThe hematologic response (HR) rate to IST in ANC reponded patients was significantly higher than non-responded group (3-month HR 49.0% vs 28.9%, P=0.023; 6-month HR 61.2% vs 40.8%, P=0.026). With ∆ANC≥0.5×10⁹/L as cutoff level, the best point to predict early IST response was 10 days after G-CSF (d 10). Response of ANC to pre-IST G-CSF treatment at d 10 was among the independent factors of predicting 3-month (P=0.004), but not for 6-month response to IST. The overall 5-year survival rate was 92.8% and 69.5% in ANC responded and non-responed groups, respectively (P=0.025).

CONCLUSIONResponding to pre-IST G-CSF treatment reflected the residual bone marrow hematopoiesis, and could act as a convenient and practical predictor to early IST response as well as long-term survival in SAA.

Anemia, Aplastic ; drug therapy ; Granulocyte Colony-Stimulating Factor ; therapeutic use ; Humans ; Immunosuppressive Agents ; therapeutic use ; Neutrophils ; drug effects

Anemia, Aplastic ; drug therapy ; immunology ; surgery ; therapy ; Bone Marrow Transplantation ; Child ; Humans ; Immunosuppressive Agents ; therapeutic use ; Treatment Outcome

OBJECTIVETo evaluate the efficacy of antithymocyte globulin (ATG) based immunosuppression therapy for childhood aplastic anemia, to reduce the adverse effects and to observe the long-term outcome.

METHODThirty-five children with aplastic anemia (AA) were enrolled in this study. Six of the cases had very severe AA (VSAA), 11 had severe AA (SAA)-I, 8 had SAA-II and 10 had moderate AA (MAA). All these patients were treated with ATG plus Cyclosporin A (CSA). The following measures were taken during the ATG therapy: infection of the patients had been controlled before ATG treatment. Comprehensive anti-allergic measures were implemented. Close attention was paid to the hemorrhage related with platelet reduction caused by ATG and severe infection of the patients.

RESULTShortly after the ATG usage, all the patients had a significant decrease of absolute peripheral lymphoblast count by more than 60 percent. With a mean follow-up time of 28 months, the total effective rate was 77.14% (27/35), significant response rate was 57.14%(20/35). There was no significant difference among VSAA, SAA and MAA groups in the response rate. Adverse reactions included the following: (1) 48.6% (17/35) patients presented mild anaphylactoid reaction during the first day of ATG treatment; (2) 42.9%(15/35) cases presented serum sickness 5 - 11 days after the last dose of ATG with a mean duration of 3.6 days, all the patients were cured effectively with methylprednisolone; (3) 25.7% (9/35) patient's peripheral blood platelet count was reduced, might be caused by ATG, to below 10 × 10(9)/L, but no patient had severe hemorrhagic complication after platelet transfusion was performed; (4) 22.9%(8/35)of patients got infection within a month after ATG therapy, including 3 cases with clinical septicemia, all the 3 cases recovered after antibiotics treatment. There was no ATG treatment-related death in this series.

CONCLUSIONATG is a very effective therapy for children with SAA and MAA. Comprehensive measures are needed to prevent and handle the side effects to avoid treatment-related death. Long-term supportive therapy and proper follow up contribute to the favourable outcomes of the patients.

Adolescent ; Anemia, Aplastic ; drug therapy ; Antilymphocyte Serum ; therapeutic use ; Child ; Child, Preschool ; Female ; Follow-Up Studies ; Humans ; Male ; Treatment Outcome