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NEJM: Donidalorsen can reduce the risk of hereditary angioedema and improve the quality of life of patients
Time of Update: 2022-04-23
Antisense oligonucleotide Donidalorsen is a potential "best-in-class" prophylactic therapy for hereditary angioedema that reduces HAE frequency and disease burden by selectively inhibiting prekallikrein .
Antisense oligonucleotide Donidalorsen is a potential "best-in-class" prophylactic therapy for hereditary angioedema that reduces HAE frequency and disease burden by selectively inhibiting prekallikrein .
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Unusual "skin disease"
Time of Update: 2022-04-23
4% of nuclear cells, and their immunophenotypes were CD33+ very few , HLADR+, CD5+ few , CD4+, CD56+, CD2+, a small amount of CD7+ , a small amount of CD303+ , a small amount of CD304+ , CD123++, the current immunophenotype information is considered to be blastic plasmatic dendritic cell tumor ;Submitted for blood pathology, bone marrow report: abnormal lymphocytes accounted for 46% ; flow cytometry: showed that abnormal cells accounted for about 56.
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NEJM: BioMarin's Phase 3 clinical trial results of AAV therapy for hemophilia released, with positive and sustained effects
Time of Update: 2022-04-23
On March 17, 2022, the results of the Phase 3 clinical trial of Valoctocogene Roxaparvovec, an AAV gene therapy developed by BioMarin Pharmaceutical for the treatment of adults with severe hemophilia A, were officially released in the New England Journal of Medicine (NEJM) .
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HAEMATOLOGICA: Excellent survival with rituximab combined with LMB-based chemotherapy in children and adolescents with primary mediastinal large B-cell lymphoma
Time of Update: 2022-04-23
EFS, event-free survival; OS, overall survival The vertical line represents the Rothman 95% confidence intervalFrom September 2001 to March 2012, the study enrolled 42 PMLBL patients, with a median age of 15 years (range 8-18 years), and 21 patients received chemotherapy plus rituximab .
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FDA Approves Legend Bio's First CAR-T Cell Therapy Product, CARVYKTI® (Cedaki Orenza)
Time of Update: 2022-04-22
CARTITUDE-1 is an ongoing Phase 1b/2, open-label, single-arm, multicenter study evaluating the safety and efficacy of CARVYKTI® in adult patients with relapsed or refractory multiple myeloma who are Patients received at least 3 lines of prior therapy, including proteasome inhibitors (PIs), immunomodulators (IMiDs), and anti-CD38 monoclonal antibodies .
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AJH: Expression of p53 protein isoforms predicts survival in patients with multiple myeloma
Time of Update: 2022-04-22
Figure 4: Simultaneous effects of cytogenetic risk and p53 protein isoform levels on progression-free and overall survival in MM patients .
Figure 4: Simultaneous effects of cytogenetic risk and p53 protein isoform levels on progression-free and overall survival in MM patients .
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[Live Preview] Draw the largest concentric circle for rare diseases International Rare Disease Day
Time of Update: 2022-04-22
Zhang Fabao, the founder of Metz Medical, will be invited to participate in the foundation inauguration ceremony, and discuss with many bigwigs in the industry to accelerate China's promotion of global rare disease research and translational medicine .
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Blood: Sirolimus + Prednisolone vs Sirolimus Monotherapy for Kaposi-like Hemangioendothelioma
Time of Update: 2022-04-22
This study is a multicenter, randomized, controlled, open-label clinical trial to compare sirolimus plus prednisolone versus sirolimus monotherapy for carboxylate with Kasabach-Merritt phenomenon Efficacy and safety of hemangioendothelioma .
7%) patients in the combination and monotherapy groups, respectively, achieved sustained platelet remission (response rate difference: 27.
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Did you know this rare form of AML with visible goblet cells?
Time of Update: 2022-04-22
This is a case report published in the British Journal of Hematology in 2018 :Two of the AML with t(6;9)(p23;q34), DEK-NUP214 cases showed typical goblet cells.
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BJH: Switching to Avambopag after Eltrombopag or Romigrastim in Adults with Immune Thrombocytopenia: A Multicenter US Study
Time of Update: 2022-04-22
Abbreviations: AVA, platelets, PLT, PLT, platelet count, ROMI,We conducted a retrospective observational study of adults with ITP who were converted from eltrombopag or romipprotine to avantgarde at four tertiary ITP referral centers in the United States .
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Nature sub-issue: Peking University Hu Jiazhi's team developed a safer Cas9 variant Cas9TX
Time of Update: 2022-04-22
Cas9TX can inhibit the occurrence of chromosomal structural abnormalities such as chromosomal translocations and large fragment deletions during gene editing, greatly improving the safety of CRISPR-Cas9 gene editing, and reducing the DNA damage during the editing process to the level currently considered the safest.
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The Influence of Hemolyzed Specimen on Biochemical Items and Handling Methods
Time of Update: 2022-04-22
Which biochemical items will be affected and its impact mechanism ?Items with high resultsItems with high results Items with high results(1) Aspartate aminotransferase (AST): The content of AST in red blood cells is nearly 40 times higher than that in plasma , so AST can be increased during hemolysis .
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Acta Haematol: Impact of chemotherapy or allogeneic stem cell transplantation as salvage therapy in patients with refractory acute myeloid leukemia
Time of Update: 2022-04-22
A total of 29 patients received allo-HSCT directly without salvage chemotherapy, and 26 patients achieved complete remission (CR) or complete remission with incomplete hematologic recovery (CRi) after transplantation, 4-year leukemia -free survival (LFS) and Overall survival (OS) was 45.
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Haematologica: Mutations in RXRA renew interest in retinoids for acute myeloid leukemia
Time of Update: 2022-04-22
In APL, a subtype that accounts for 5% of AML cases, a combination of two targeted agents, all-trans retinoic acid (ATRA) and arsenic trioxide, cured more than 90% of AML through PML-RARA driver degradation, differentiation and recovery Patients with PML-dependent aging .
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Crit Care: Impact of platelet transfusion on outcomes in trauma patients
Time of Update: 2022-04-22
The purpose of this study was to 1) evaluate the effect of early platelet transfusion on 24-hour all-cause mortality; 2) describe the platelet count at admission and its Relationship between trauma severity and patient outcomes .
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AJH: Decitabine and Vorinostat in Combination with FLAG Chemotherapy for Pediatric Relapsed/Refractory AML: A Treatment Progress Report from the Childhood Leukemia and Lymphoma (TACL) Consortium
Time of Update: 2022-04-22
Decitabine and vorinostat in combination with FLAG chemotherapy is well tolerated, biologically active, and effective in pediatric patients with R/R AML, especially those with epigenetic alterations Original source:Original source:Pommert, L.
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Clin Cancer Res: Decitabine combined with PARP inhibitor tazopanib for refractory/relapsed acute myeloid leukemia
Time of Update: 2022-04-22
explored the recommended Phase 2 dose, initial activity and safety of DNMTi decitabine in combination with PARPi tazopanib in patients with relapsed or refractory AML .
In conclusion, the combination of decitabine/tazopanib is well tolerated in patients with relapsed/refractory AML, and has preliminary antitumor activity , which deserves further study .
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Leukemia: Rotecept for myelodysplastic syndromes/myeloproliferative neoplasms with ring sideroblasts and thrombocythemia
Time of Update: 2022-04-22
Numerical values were higher in patients with high transfusion burden (≥4 units/8 weeks) randomized to rotacept versus placebo, and RBC-TI ≥8 weeks (4/8 [50.
1% of MDS/MPN-RS-T patients randomized to rotercept and placebo achieved mHI-E, compared with 52.
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How should previously treated severe hemophilia A be treated?
Time of Update: 2022-04-22
First-line results showed clinically meaningful prevention of bleeding events in patients receiving weekly prophylaxis over 52 weeks, with an average annual bleeding rate (ABR) of 0.
First-line results showed clinically meaningful prevention of bleeding events in patients receiving weekly prophylaxis over 52 weeks, with an average annual bleeding rate (ABR) of 0.
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Blood: Effects of FLT3 inhibitor Gilteritinib vs salvage chemotherapy on long-term outcomes in patients with relapsed/refractory AML
Time of Update: 2022-04-22
Chemotherapy combination) significantly improved the survival rate and remission rate of AML patientsThe randomized phase 3 ADMIRAL trial compared gilteritinib with salvage chemotherapy in patients with FLT3 mutation-positive (+) relapsed or refractory AML .
