Efficacy, Safety and Quality of Life of Pegcetacoplan in Japanese Patients With Paroxysmal Nocturnal Hemoglobinuria Treated Within the Phase 3 PEGASUS Trial
CONCLUSION: These data suggest that Japanese patients with PNH can be effectively and safely managed with pegcetacoplan.CLINICALTRIALS: gov identifier: NCT03500549.PMID:38615657 | DOI:10.1159/000537696 (Source: Acta Haematologica)
Source: Acta Haematologica - April 14, 2024 Category: Hematology Authors: Hisakazu Nishimori Hideyuki Nakazawa Shinobu Tamura Toshiki Uchida Kensuke Usuki Johan Szamosi R égis Peffault de Latour Alexander R öth Jens Panse Source Type: research
Long-term efficacy and safety of damoctocog alfa pegol prophylaxis in patients with haemophilia A aged 12- & lt;18 years at enrolment into PROTECT VIII
Conclusion Efficacy and safety of damoctocog alfa pegol was confirmed in adolescent patients with haemophilia A, with data for up to 6 years supporting its use as a long-term treatment option in this group as they transition into adulthood.PMID:38599195 | DOI:10.1159/000538702 (Source: Acta Haematologica)
Source: Acta Haematologica - April 10, 2024 Category: Hematology Authors: Mark T Reding Mindy Simpson Jonathan Ducore P ål Andrè Holme Monika Maas Enriquez Maria Elisa Mancuso Source Type: research
Long-term efficacy and safety of damoctocog alfa pegol prophylaxis in patients with haemophilia A aged 12- & lt;18 years at enrolment into PROTECT VIII
Conclusion Efficacy and safety of damoctocog alfa pegol was confirmed in adolescent patients with haemophilia A, with data for up to 6 years supporting its use as a long-term treatment option in this group as they transition into adulthood.PMID:38599195 | DOI:10.1159/000538702 (Source: Acta Haematologica)
Source: Acta Haematologica - April 10, 2024 Category: Hematology Authors: Mark T Reding Mindy Simpson Jonathan Ducore P ål Andrè Holme Monika Maas Enriquez Maria Elisa Mancuso Source Type: research
Long-term efficacy and safety of damoctocog alfa pegol prophylaxis in patients with haemophilia A aged 12- & lt;18 years at enrolment into PROTECT VIII
Conclusion Efficacy and safety of damoctocog alfa pegol was confirmed in adolescent patients with haemophilia A, with data for up to 6 years supporting its use as a long-term treatment option in this group as they transition into adulthood.PMID:38599195 | DOI:10.1159/000538702 (Source: Acta Haematologica)
Source: Acta Haematologica - April 10, 2024 Category: Hematology Authors: Mark T Reding Mindy Simpson Jonathan Ducore P ål Andrè Holme Monika Maas Enriquez Maria Elisa Mancuso Source Type: research
Long-term efficacy and safety of damoctocog alfa pegol prophylaxis in patients with haemophilia A aged 12- & lt;18 years at enrolment into PROTECT VIII
Conclusion Efficacy and safety of damoctocog alfa pegol was confirmed in adolescent patients with haemophilia A, with data for up to 6 years supporting its use as a long-term treatment option in this group as they transition into adulthood.PMID:38599195 | DOI:10.1159/000538702 (Source: Acta Haematologica)
Source: Acta Haematologica - April 10, 2024 Category: Hematology Authors: Mark T Reding Mindy Simpson Jonathan Ducore P ål Andrè Holme Monika Maas Enriquez Maria Elisa Mancuso Source Type: research
Failure of treatment-free remission after a prolonged deep molecular response in patients with chronic myeloid leukemia
Acta Haematol. 2024 Apr 4. doi: 10.1159/000538651. Online ahead of print.ABSTRACTPatients with chronic myeloid leukemia in chronic phase (CML-CP) can have a normal life expectancy when treated with the BCR::ABL1 tyrosine kinase inhibitors. In recent years, treatment discontinuation and treatment-free remission (TFR) emerged as the new goal of therapy in patients with CML-CP. Deep and sustained molecular remissions for more than 3 to 5 years are associated with higher chances of a successful TFR. However, although uncommon, some patients may still experience molecular or hematological relapse after treatment discontinuation...
Source: Acta Haematologica - April 4, 2024 Category: Hematology Authors: Patrice E Nasnas Elias J Jabbour Koji Sasaki Ghayas C Issa Lucia Masarova Nicholas J Short Fadi G Haddad Source Type: research
Failure of treatment-free remission after a prolonged deep molecular response in patients with chronic myeloid leukemia
Acta Haematol. 2024 Apr 4. doi: 10.1159/000538651. Online ahead of print.ABSTRACTPatients with chronic myeloid leukemia in chronic phase (CML-CP) can have a normal life expectancy when treated with the BCR::ABL1 tyrosine kinase inhibitors. In recent years, treatment discontinuation and treatment-free remission (TFR) emerged as the new goal of therapy in patients with CML-CP. Deep and sustained molecular remissions for more than 3 to 5 years are associated with higher chances of a successful TFR. However, although uncommon, some patients may still experience molecular or hematological relapse after treatment discontinuation...
Source: Acta Haematologica - April 4, 2024 Category: Hematology Authors: Patrice E Nasnas Elias J Jabbour Koji Sasaki Ghayas C Issa Lucia Masarova Nicholas J Short Fadi G Haddad Source Type: research
Failure of treatment-free remission after a prolonged deep molecular response in patients with chronic myeloid leukemia
Acta Haematol. 2024 Apr 4. doi: 10.1159/000538651. Online ahead of print.ABSTRACTPatients with chronic myeloid leukemia in chronic phase (CML-CP) can have a normal life expectancy when treated with the BCR::ABL1 tyrosine kinase inhibitors. In recent years, treatment discontinuation and treatment-free remission (TFR) emerged as the new goal of therapy in patients with CML-CP. Deep and sustained molecular remissions for more than 3 to 5 years are associated with higher chances of a successful TFR. However, although uncommon, some patients may still experience molecular or hematological relapse after treatment discontinuation...
Source: Acta Haematologica - April 4, 2024 Category: Hematology Authors: Patrice E Nasnas Elias J Jabbour Koji Sasaki Ghayas C Issa Lucia Masarova Nicholas J Short Fadi G Haddad Source Type: research
Failure of treatment-free remission after a prolonged deep molecular response in patients with chronic myeloid leukemia
Acta Haematol. 2024 Apr 4. doi: 10.1159/000538651. Online ahead of print.ABSTRACTPatients with chronic myeloid leukemia in chronic phase (CML-CP) can have a normal life expectancy when treated with the BCR::ABL1 tyrosine kinase inhibitors. In recent years, treatment discontinuation and treatment-free remission (TFR) emerged as the new goal of therapy in patients with CML-CP. Deep and sustained molecular remissions for more than 3 to 5 years are associated with higher chances of a successful TFR. However, although uncommon, some patients may still experience molecular or hematological relapse after treatment discontinuation...
Source: Acta Haematologica - April 4, 2024 Category: Hematology Authors: Patrice E Nasnas Elias J Jabbour Koji Sasaki Ghayas C Issa Lucia Masarova Nicholas J Short Fadi G Haddad Source Type: research
Failure of treatment-free remission after a prolonged deep molecular response in patients with chronic myeloid leukemia
Acta Haematol. 2024 Apr 4. doi: 10.1159/000538651. Online ahead of print.ABSTRACTPatients with chronic myeloid leukemia in chronic phase (CML-CP) can have a normal life expectancy when treated with the BCR::ABL1 tyrosine kinase inhibitors. In recent years, treatment discontinuation and treatment-free remission (TFR) emerged as the new goal of therapy in patients with CML-CP. Deep and sustained molecular remissions for more than 3 to 5 years are associated with higher chances of a successful TFR. However, although uncommon, some patients may still experience molecular or hematological relapse after treatment discontinuation...
Source: Acta Haematologica - April 4, 2024 Category: Hematology Authors: Patrice E Nasnas Elias J Jabbour Koji Sasaki Ghayas C Issa Lucia Masarova Nicholas J Short Fadi G Haddad Source Type: research
Consensus-Based Expert Recommendations on the Management of Hemophilia A in the Gulf Region
Acta Haematol. 2024 Apr 2. doi: 10.1159/000538400. Online ahead of print.ABSTRACTBACKGROUND: Hemophilia A presents a significant health challenge in the Gulf region, where it has an especially high prevalence. There are several unmet needs associated with the management of hemophilia A in the region. The aim of this manuscript is to contextualize unmet management needs, provide recommendations to optimize care, and specify requirements for the establishment of gene therapy centers in the region.SUMMARY: An expert panel was assembled comprising ten clinical hematologists from Kuwait, Oman, Saudi Arabia, and the United Arab ...
Source: Acta Haematologica - April 2, 2024 Category: Hematology Authors: Hazzaa Abdulla Alzahrani Ashraf Warsi Ali Mullah-Ali Ghazi Alotaibi Mahmoud Abu-Riash Mohammad Alshahrani Mustaqeem Siddiqui Tarek Owaidah Shahrukh Hashmi Source Type: research
Consensus-Based Expert Recommendations on the Management of Hemophilia A in the Gulf Region
Acta Haematol. 2024 Apr 2. doi: 10.1159/000538400. Online ahead of print.ABSTRACTBACKGROUND: Hemophilia A presents a significant health challenge in the Gulf region, where it has an especially high prevalence. There are several unmet needs associated with the management of hemophilia A in the region. The aim of this manuscript is to contextualize unmet management needs, provide recommendations to optimize care, and specify requirements for the establishment of gene therapy centers in the region.SUMMARY: An expert panel was assembled comprising ten clinical hematologists from Kuwait, Oman, Saudi Arabia, and the United Arab ...
Source: Acta Haematologica - April 2, 2024 Category: Hematology Authors: Hazzaa Abdulla Alzahrani Ashraf Warsi Ali Mullah-Ali Ghazi Alotaibi Mahmoud Abu-Riash Mohammad Alshahrani Mustaqeem Siddiqui Tarek Owaidah Shahrukh Hashmi Source Type: research
Long Non-coding RNA RP11-252C15.1 is a Potential Biomarker of Prognosis and Hallmark for Leukemogenesis in Children with B-cell Precursor Acute Lymphoblastic Leukemia
CONCLUSION: LncRNA RP11-252C15.1 is a potential oncogene in BCP-ALL pathogenesis and a prognostic biomarker in children with BCP-ALL.PMID:38527424 | DOI:10.1159/000535461 (Source: Acta Haematologica)
Source: Acta Haematologica - March 25, 2024 Category: Hematology Authors: Lili Pan Yongzhi Zheng Hao Zheng Source Type: research
The ratio of serum urea nitrogen to albumin is a better predictor of OS in MM patients than urea nitrogen alone
Conclusion This retrospective study based on the MMRF database showed that high sUN and sUAR levels were positively associated with β2-MG, LDH, and ISS staging, and sUAR exhibited a stronger correlation with OS than sUN alone.PMID:38527425 | DOI:10.1159/000538479 (Source: Acta Haematologica)
Source: Acta Haematologica - March 25, 2024 Category: Hematology Authors: Jiaqi Shao Enfan Zhang Haoguang Chen Zhen Cai Mengmeng Dong Source Type: research
Utility of echocardiogram in neutropenic patients with gram positive bacteremia - a retrospective study
Conclusion In our experience, the yield of echocardiogram in hemato-oncological neutropenic patients with bacteremia is extremely low, owing to reduced probability of IE in this population, and thus could be avoided in most cases.PMID:38527433 | DOI:10.1159/000538449 (Source: Acta Haematologica)
Source: Acta Haematologica - March 25, 2024 Category: Hematology Authors: Neta Sternbach Haim Ben-Zvi Ofir Wolach Moshe Yeshurun Pia Raanani Dafna Yahav Liat Shargian Source Type: research
