Clinical characterization of hemophagocytic lymphohistiocytosis caused by immune checkpoint inhibitors: a review of published cases
CONCLUSION: HLH should be not underestimated as a potentially serious adverse effect of immune checkpoint inhibitors since appropriate treatments may save the life of patients.PMID:38606818 | DOI:10.1080/16078454.2024.2340144 (Source: Hematology)
Source: Hematology - April 12, 2024 Category: Hematology Authors: Zhiya Xu Huilan Li Xinyi Yu Jia Luo Zanling Zhang Source Type: research

Combination therapy with venetoclax and azacitidine for the treatment of myelodysplastic syndromes with < em > DDX41 < /em > mutations
In this study, we evaluated the efficacy of venetoclax and azacitidine combination therapy in eight consecutive MDS patients with DDX41 mutations at our centre from March 2021 to November 2023. We retrospectively analyzed the genetic features and clinical characteristics of these patients. Our findings suggest that MDS patients with DDX41 mutation may benefit from the therapy, for six subjects received this regimen as initial therapy and five of the six subjects achieved complete remission.PMID:38597818 | DOI:10.1080/16078454.2024.2338509 (Source: Hematology)
Source: Hematology - April 10, 2024 Category: Hematology Authors: Xin Wang Zhijian Xiao Tiejun Qin Zefeng Xu Yujiao Jia Shiqiang Qu Bing Li Lijuan Pan Qingyan Gao Meng Jiao Robert Peter Gale Source Type: research

Donor-type bone marrow aplasia following hematopoietic stem cell transplantation in a child with a novel < em > SAMD9L < /em > variant
We report a case of a previously healthy, 3-year-old boy with no dysmorphology, who presented with severe aplastic anemia and a novel variant in the SAMD9L gene. His father, elder brother and sister who harbored the same variant were completely healthy. In the absence of a matched unrelated donor, he underwent a stem cell transplant from his sister, a 10/10 match. Almost 2 years later he developed donor type aplasia and succumbed to an invasive fungal infection after a failed haplograft from his mother. This case highlights the pathogenicity of this previously undescribed germline variation of uncertain significance in the...
Source: Hematology - April 10, 2024 Category: Hematology Authors: Manujasri Wimalachandra Ruwangi Dissanayake Revathi Raj Austin Kulasekeraraj Sujith Samarasinghe Lallindra Gooneratne Source Type: research

Post-transplant cyclophosphamide or cell selection in haploidentical allogeneic hematopoietic cell transplantation?
DISCUSSION: While acknowledging that no randomized controlled prospective studies have been yet conducted comparing TCD versus PTCy in haploidentical allo-HCT recipients, there are two advantages that would favor the PTCy, namely ease of application and lower cost. However, emerging data on adverse events associated with PTCy including, but not limited to cardiac associated toxicities or increased incidence of post-allograft infections, and others, are important to recognize.PMID:38597828 | DOI:10.1080/16078454.2024.2326384 (Source: Hematology)
Source: Hematology - April 10, 2024 Category: Hematology Authors: Razan Mohty Zaid Al Kadhimi Mohamed Kharfan-Dabaja Source Type: research

Combination therapy with venetoclax and azacitidine for the treatment of myelodysplastic syndromes with < em > DDX41 < /em > mutations
In this study, we evaluated the efficacy of venetoclax and azacitidine combination therapy in eight consecutive MDS patients with DDX41 mutations at our centre from March 2021 to November 2023. We retrospectively analyzed the genetic features and clinical characteristics of these patients. Our findings suggest that MDS patients with DDX41 mutation may benefit from the therapy, for six subjects received this regimen as initial therapy and five of the six subjects achieved complete remission.PMID:38597818 | DOI:10.1080/16078454.2024.2338509 (Source: Hematology)
Source: Hematology - April 10, 2024 Category: Hematology Authors: Xin Wang Zhijian Xiao Tiejun Qin Zefeng Xu Yujiao Jia Shiqiang Qu Bing Li Lijuan Pan Qingyan Gao Meng Jiao Robert Peter Gale Source Type: research

Donor-type bone marrow aplasia following hematopoietic stem cell transplantation in a child with a novel < em > SAMD9L < /em > variant
We report a case of a previously healthy, 3-year-old boy with no dysmorphology, who presented with severe aplastic anemia and a novel variant in the SAMD9L gene. His father, elder brother and sister who harbored the same variant were completely healthy. In the absence of a matched unrelated donor, he underwent a stem cell transplant from his sister, a 10/10 match. Almost 2 years later he developed donor type aplasia and succumbed to an invasive fungal infection after a failed haplograft from his mother. This case highlights the pathogenicity of this previously undescribed germline variation of uncertain significance in the...
Source: Hematology - April 10, 2024 Category: Hematology Authors: Manujasri Wimalachandra Ruwangi Dissanayake Revathi Raj Austin Kulasekeraraj Sujith Samarasinghe Lallindra Gooneratne Source Type: research

Post-transplant cyclophosphamide or cell selection in haploidentical allogeneic hematopoietic cell transplantation?
DISCUSSION: While acknowledging that no randomized controlled prospective studies have been yet conducted comparing TCD versus PTCy in haploidentical allo-HCT recipients, there are two advantages that would favor the PTCy, namely ease of application and lower cost. However, emerging data on adverse events associated with PTCy including, but not limited to cardiac associated toxicities or increased incidence of post-allograft infections, and others, are important to recognize.PMID:38597828 | DOI:10.1080/16078454.2024.2326384 (Source: Hematology)
Source: Hematology - April 10, 2024 Category: Hematology Authors: Razan Mohty Zaid Al Kadhimi Mohamed Kharfan-Dabaja Source Type: research

A review of immunotargeted therapy for Philadelphia chromosome positive acute lymphoblastic leukaemia: making progress in chemotherapy-free regimens
Hematology. 2024 Dec;29(1):2335856. doi: 10.1080/16078454.2024.2335856. Epub 2024 Apr 6.ABSTRACTPhiladelphia chromosome-positive acute lymphoblastic leukemia (PH + ALL) is the most common cytogenetic abnormality of B-ALL in adults and is associated with poor prognosis. Previously, the only curative treatment option in PH + ALL was allogeneic hematopoietic stem cell transplantation (Allo-HSCT). Since 2000, targeted therapy combined with chemotherapy, represented by the tyrosine kinase inhibitor Imatinib, has become the first-line treatment for PH + ALL. Currently, the remission rate and survival rate of Imatinib are superio...
Source: Hematology - April 6, 2024 Category: Hematology Authors: Zhen-Yu Xiong Yao-Jia Shen Shi-Zhong Zhang Hong-Hu Zhu Source Type: research

Epidemiological characteristics and influencing factors of acute leukemia in children and adolescents and adults: a large population-based study
CONCLUSION: From 2000 to 2016, the incidence rates of AL in children and adolescents and adults were increasing. Children and adolescents with AL had significantly better OS than adults with AL, and OS declined with age in both children and adolescents and adults with ALL, AML and AUL.PMID:38578123 | DOI:10.1080/16078454.2024.2327916 (Source: Hematology)
Source: Hematology - April 5, 2024 Category: Hematology Authors: Shuojie Liu Bin Hu Jiaqin Zhang Source Type: research

Honokiol induces apoptosis and autophagy in dexamethasone-resistant T-acute lymphoblastic leukemia CEM-C1 cells
Conclusion: The combination of honokiol and DEX were better than DEX alone in DEX-resistant CEM-C1 cell lines. Honokiol may regulate T-ALL-related dexamethasone resistance by affecting c-Myc.PMID:38573223 | DOI:10.1080/16078454.2024.2337307 (Source: Hematology)
Source: Hematology - April 4, 2024 Category: Hematology Authors: Yang Liu Suqian Zhang Yajuan Tan Source Type: research