Child ; Erythropoietin ; adverse effects ; Humans ; Male ; Recombinant Proteins ; Red-Cell Aplasia, Pure ; chemically induced

Gaucher disease is a lysosomal storage disease for which enzyme replacement therapy has proven to be effective. A switch-over clinical trial was performed to evaluate the efficacy and safety of Abcertin(R) (ISU Abxis, Seoul, Korea) in subjects with type 1 Gaucher disease who were previously treated with imiglucerase. Five Korean patients with type 1 Gaucher disease were enrolled. Previous doses of imiglucerase ranged from 30 to 55 U/kg every other week. The same dose of Abcertin(R) was administered to all patients for 24 weeks. Primary efficacy endpoints were changes in hemoglobin levels and platelet counts, and the secondary efficacy endpoints included changes in liver and spleen volumes, serum biomarkers, skeletal status and bone mineral density (BMD). During the study period, no statistically significant changes were observed in all parameters including hemoglobin levels and platelet counts, liver and spleen volumes, skeletal status and BMD. Abcertin(R) administration was continued in three patients for another 24 weeks as an extension of the study. Hemoglobin levels and platelet counts were maintained in all three patients. In conclusion, the efficacy and safety of Abcertin(R) are similar to those of imiglucerase, and Abcertin(R) is an effective therapeutic agent for patients with type 1 Gaucher disease (Clinical Trial Registry No. NCT02053896 at www.clinicaltrials.gov).
Adolescent ; Adult ; Biosimilar Pharmaceuticals/adverse effects/pharmacokinetics/*therapeutic use ; Child ; *Enzyme Replacement Therapy/adverse effects ; Female ; Gaucher Disease/blood/*drug therapy ; Glucosylceramidase/adverse effects/pharmacokinetics/*therapeutic use ; Humans ; Male ; Recombinant Proteins/adverse effects/pharmacokinetics/*therapeutic use

Adult ; Antiviral Agents ; adverse effects ; therapeutic use ; Drug Therapy, Combination ; Female ; Hearing Disorders ; chemically induced ; Hepatitis C, Chronic ; drug therapy ; Humans ; Interferon-alpha ; adverse effects ; therapeutic use ; Middle Aged ; Recombinant Proteins ; adverse effects ; therapeutic use ; Ribavirin ; adverse effects ; therapeutic use

Tn order to set up a mouse model of myelofibrosis (MF) induced with high dose recombinant human erythropoietin (rhEPO). 60 mice were collected and divided into EPO and control groups, the former was injected with rhEPO and the latter with normal saline intraperitoneally. 5 mice from each group were executed on day 6, 30, 60, 90, 120 and 150 respectively. Their WBC count, Hb level, MCV, RDW and platelet amount were measured by automatic blood cell analyzer; CD34(+) cell ratio in bone marrow were analyzed by flow cytometry; liver and spleen coefficients were measured; pathological changes of liver, spleen, femur were observed by HE staining and reticular fibers staining; cortex thickness, femoral canal diameter and lumbar spine density were determined by computerized tomography (CT). The results indicated that as compared with normal control group in EPO induced group, WBC count was increased slightly in whole period, but without statistic significance (p > 0.05), Hb level and RDW increased at day 6 and 30 significantly (p < 0.05), MCV increased at day 6 significantly (p < 0.05), but platelet amount decreased significantly at all time points (p < 0.05). Most mice in EPO-induced group had hepatomegalia and their liver and spleen coefficient increased significantly at day 60 (p < 0.05), while most mice had splenomegaly and its coefficient was increased significantly at all time-points (p < 0.05). CD43(+) cell ratio of EPO group increased significantly in whole period (p < 0.05). CT scanning displayed femoral cortical thickening, medulla canal narrowing and lumbar spine density increasing at day 150, meanwhile, HE staining and reticular fiber staining showed the fatty degeneration or vacuolization in liver, splenomegaly with megakaryocytic proliferation, femur bone marrow fibrosis and osteosclerosis. It is concluded that the mouse induced by high dose of rhEPO displays the myelofibrosis associated with splenic extramedullary hemopoiesis, and this study is useful to establish a practical MF model, and to explore its pathological mechanism.
Animals ; Disease Models, Animal ; Erythropoietin ; adverse effects ; Female ; Humans ; Mice ; Mice, Inbred Strains ; Primary Myelofibrosis ; chemically induced ; Recombinant Proteins ; adverse effects

Myelosupression is the major dose-limiting toxicity in most chemotherapeutic drugs. To evaluate the curative effect of a domestic product of rhG-CSF on chemotherapy-induced leukopenia, 132 patients with malignancies were enrolled, including 80 patients with lung cancer, 35 patients with breast cancer, 10 patients with nasopharyngeal carcinoma, 3 patients with non-Hodgkin's lymphoma, 2 patients with gastric carcinoma and 2 patients with bone metastasis. Total of 528 cycles of chemotherapy were performed. The results showed that according the grade of leukopenia, the different daily doses of the domestic product of rhG-CSF were administered: 75 micro g/d for 3 days, 150 micro g/d for 4 days and 300 micro g/d for 5 days, in grade I-II, grade III and grade IV groups, respectively, the times of recovery to normal level of white blood cells were 2.5, 4.2 and 7 days in 3 groups, respectively. In conclusion, The Chinese product of rhG-CSF shortened the duration of leukopenia and accelerated the hematologic recovery, which shows only slight side effects, so that patients receive the optimal doses of chemotherapy and completed the planned schedule on time.
Adolescent ; Adult ; Aged ; Antineoplastic Agents ; adverse effects ; Female ; Granulocyte Colony-Stimulating Factor ; adverse effects ; therapeutic use ; Humans ; Leukopenia ; drug therapy ; Male ; Middle Aged ; Neoplasms ; drug therapy ; Recombinant Proteins

Recombinant human growth hormone is generally safe in treating children with growth hormone deficiency and idiopathic short stature. However, side effects such as sodium and water retention, benign intracranial hypertension, insulin insensitivity, increasing risk of secondary neoplasm, scoliosis, and slipped capital femoral epiphysis may occur occasionally, although the overall incidence remains low.
Dwarfism ; drug therapy ; Dwarfism, Pituitary ; drug therapy ; Human Growth Hormone ; adverse effects ; deficiency ; therapeutic use ; Humans ; Recombinant Proteins ; adverse effects ; therapeutic use

This study was to evaluate the effect of a Chinese genetic engineering product of rhG-CSF (Lishengsu) on leukopenia induced by chemoradiotherapy in patients with malignant solid tumors. One hundred and forty cases of leukopenia following chemoradiotherapy for malignant tumors were treated with Lishengsu. When the total leukocyte counts (WBC) less than 3.0 x 10(9)/L or the absolute neutrophil count (ANC) less than 2.0 x 10(9)/L, Lishengsu was given 75 micro g/day subantaneously and the administration was stopped when the WBC counts rose up to 4.0 x 10(9)/L or higher, or the ANC counts reached 2.5 x 10(9)/L or more. The results showed that peripheral leukocyte counts in all patients following the treatment with Lishengsu were recovered and the average time of its administration was 4.8 days. The rate of its efficaciousness was 96.4%. It is concluded that Lishengsu could elevate nadirs of leukopenia and significantly shortened time period of leukopenia below 4.0 x 10(9)/L with acceptable mild side effects, decrease complications of infection so that the chinese product of rhG-CSF facilitates chemoradiotherapy significantly.
Adult ; Aged ; Antineoplastic Agents ; adverse effects ; Female ; Granulocyte Colony-Stimulating Factor ; adverse effects ; therapeutic use ; Humans ; Leukopenia ; drug therapy ; Male ; Middle Aged ; Neoplasms ; complications ; drug therapy ; Recombinant Proteins

This study was purposed to evaluate the effects of recombinant human granulocyte colony-stimulating factor (rhG-CSF) on hematopoietic reconstruction and survival in beagles exposed to mixed fission neutron and γ-ray. 13 beagles were unilaterally exposed to single dose of 2.3 Gy 90% neutrons. The experiments were divided into 3 groups: irradiation control group (no any treatment, n = 4), supportive care group (n = 5) and rhG-CSF plus supportive care group (n = 4, abbreviated as rhG-CSF group) in which the beagles were subcutaneously injected with 200 µg/kg of rhG-CSF early at half an hour and 24 hours post-irradiation respectively. The results showed that 2.3 Gy 90% neutron irradiation induced a severe acute radiation sickness of bone marrow type. The administration of rhG-CSF increased the survival rate from 60% in supportive care group to 100%. Twice injection of rhG-CSF in the first 24 hours reduced duration of neutropenia, enhanced neutrophil nadir and promoted neutrophil recovery when compared with control cohort administered clinical support. The number of colony-forming cells (CFU-GM, CFU-E, and BFU-E) in peripheral blood of rhG-CSF treated canines increased 2-to 5-fold relative to those of the supportive care group on day 3. All canines treated with rhG-CSF achieved hematopoietic reconstruction as evidenced by the pathological section of sternum while severe shortage of hemopoietic cells remained in the cohorts given supportive care alone. It is concluded that the combination of supportive care and high-dose rhG-CSF can accelerate hematopoietic recovery and enhance survival of dogs exposed to 2.3 Gy mixed neutron and gamma ray.
Animals ; Dogs ; Gamma Rays ; adverse effects ; Granulocyte Colony-Stimulating Factor ; administration & dosage ; pharmacology ; Hematopoietic System ; drug effects ; radiation effects ; Neutron Diffraction ; Recombinant Proteins ; administration & dosage ; pharmacology ; Survival Rate

BACKGROUND: Recombinant human thrombopoietin (rh-TPO) and eltrombopag are two distinct TPO receptor agonists (TPO-RAs) with different mechanisms. During the pandemic, when immunosuppressive medications are controversial, switching to another TPO-RA may be worth exploring in patients who do not benefit from their first TPO-RA. We investigated the outcomes of switching from rh-TPO to eltrombopag or vice versa in immune thrombocytopenia (ITP) patients. METHODS: This prospective, open-label, observational investigation included 96 adult ITP patients who needed to switch between rh-TPO and eltrombopag between January 2020 and January 2021 at Peking University People's Hospital in China. The study evaluated response rates and platelet counts at different time points after the switch, bleeding events, time to response, duration of response, and adverse events. RESULTS: At 6 weeks after switching, response was observed in 21/49 patients (43%) who switched for inefficacy and 34/47 patients (72%) who switched for non-efficacy-related issues. In the inefficacy group, 9/27 patients (33%) responded to eltrombopag, and 12/22 patients (55%) responded to rh-TPO. In the non-efficacy-related group, 21/26 (81%) and 13/21 (62%) patients in the eltrombopag and rh-TPO groups maintained their response rates at 6 weeks after switching, respectively. Response at 6 months was achieved in 24/49 patients (49%) switching for inefficacy and 37/47 patients (79%) switching for non-efficacy issues. In the inefficacy group, 13/27 patients (48%) responded to eltrombopag, and 11/22 patients (50%) responded to rh-TPO. In the non-efficacy-related group, 22/26 patients (85%) and 15/21 patients (71%) in the eltrombopag and rh-TPO groups maintained their response rates at 6 months after switching, respectively. Both eltrombopag and rh-TPO were well tolerated. CONCLUSIONS: Our study confirmed the safety and effectiveness of switching between rh-TPO and eltrombopag for ITP patients who had no response to or experienced adverse events with their first TPO-RA. When the switch was motivated by other reasons, including patient preference and platelet count fluctuations, the probability of response was high. REGISTRATION ClinicalTrials.gov, NCT04214951.
Adult ; Humans ; Purpura, Thrombocytopenic, Idiopathic ; Thrombopoietin/adverse effects* ; Prospective Studies ; Recombinant Fusion Proteins ; Receptors, Fc/therapeutic use* ; Receptors, Thrombopoietin/therapeutic use* ; Thrombocytopenia/chemically induced* ; Benzoates/adverse effects* ; Hydrazines/adverse effects*

Aged, 80 and over ; Carcinoma, Renal Cell ; drug therapy ; Female ; Humans ; Interferon-alpha ; adverse effects ; therapeutic use ; Interleukin-2 ; adverse effects ; therapeutic use ; Lung Diseases, Interstitial ; chemically induced ; diagnosis ; Recombinant Proteins ; adverse effects ; therapeutic use