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Fulltext Luspatercept-induced reduction in transfusion requirement in α-thalassemia

Jackson N, Khairullah S, Bee PC

EJHaem, 2020 Jul;1(1):297-299.
PMID: 35847726 DOI: 10.1002/jha2.54
Fulltext In-hospital mortality in SARS-CoV-2 stratified by hemoglobin levels: A retrospective study

Al-Jarallah M, Rajan R, Saber AA, Pan J, Al-Sultan AT, Abdelnaby H, et al.

EJHaem, 2021 Aug;2(3):335-339.
PMID: 34226901 DOI: 10.1002/jha2.195

This study is to estimate in-hospital mortality in severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) patients stratified by hemoglobin (Hb) level. Patients were stratified according to hemoglobin level into two groups, that is, Hb <100 g/L and Hb >100 g/L. A total of 6931 patients were included. Of these, 6377 (92%) patients had hemoglobin levels >100 g/L. The mean age was 44 ± 17 years, and 66% of the patients were males. The median length of overall hospital stay was 13 days [2; 31]. The remaining 554 (8%) patients had a hemoglobin level <100 g/L. Overall mortality was 176 patients (2.54%) but was significantly higher in the group with hemoglobin levels <100 g/L (124, 22.4%) than in the group with hemoglobin levels >100 g/L (52, 0.82%). Risk factors associated with increased mortality were determined by multivariate analysis. The Kaplan-Meier survival analysis showed hemoglobin as a predictor of mortality. Cox proportional hazards regression coefficients for hemoglobin for the HB ≤ 100 category of hemoglobin were significant, B = 2.79, SE = 0.17, and HR = 16.34, p
Fulltext A phase III, randomised, double-blind, multi-national clinical trial comparing SB12 (proposed eculizumab biosimilar) and reference eculizumab in patients with paroxysmal nocturnal haemoglobinuria

Jang JH, Gomez RD, Bumbea H, Nogaieva L, Wong LLL, Lim SM, et al.

EJHaem, 2023 Feb;4(1):26-36.
PMID: 36819188 DOI: 10.1002/jha2.632

Treatment of paroxysmal nocturnal haemoglobinuria (PNH) includes the monoclonal antibody eculizumab. This randomised, double-blind, multi-national cross-over Phase III study in PNH patients aimed to demonstrate the equivalence of the proposed eculizumab biosimilar SB12 and reference eculizumab (Soliris, ECU). PNH patients with lactate dehydrogenase (LDH) ≥1·5× upper limit of normal were randomised into treatment sequences SB12-ECU or ECU-SB12. Four weekly infusions of 600 mg eculizumab were followed by fortnightly infusions of 900 mg until week 50 (ECU/SB12 cross-over at week 26). Primary endpoints were LDH at week 26 and the time-adjusted area under the effect curve (AUEC) of LDH over weeks 14‒26 and 40‒52. Among 46 patients (92%) who completed the study, the least squares mean (LSM) difference in LDH at week 26 (34·48; 95% confidence interval [CI] -47·66‒116·62 U/l) and geometric LSM ratio of time-adjusted AUEC of LDH (1·08; 90% CI 0·95‒1·23) were within pre-defined equivalence margins. Mean numbers of transfused red blood cell units, other secondary endpoints, pharmacokinetics, and pharmacodynamics were comparable. No patients developed anti-drug antibodies. Treatment-emergent adverse events were reported in 72% and 68% of patients in the SB12 and ECU treatment groups, respectively. The results demonstrate equivalence of SB12 to ECU and support SB12-use in PNH patients.
Clinical and haematological characteristics of 38 individuals with Hb G-Makassar in Malaysia

Esa E, Mohamad AS, Hamzah R, Hamid FSA, Aziz NA, Sevaratnam V, et al.

EJHaem, 2023 Nov;4(4):940-948.
PMID: 38024609 DOI: 10.1002/jha2.750

Haemoglobin (Hb) G-Makassar is a rare Hb variant. It presents a diagnostic challenge as it imitates sickle Hb (Hb S) in standard electrophoresis and high-performance liquid chromatography assays requiring DNA analysis to confirm diagnosis. Both have point mutations in codon 6, exon 1 in the β-globin (HBB) gene with different pathogenicities. This study describes the clinical phenotype, haematology and genotype of Hb G-Makassar. Clinical and laboratory data of 38 cases of Hb G-Makassar over 8 years were analysed. Hb G-Makassar was confirmed by a direct sequencing of HBB gene and co-inheritance of α-thalassaemia determined through multiplex gap-PCR and multiplex Amplification Refractory Mutation System polymerase chain reaction. All cases were Malays, predominantly from Terengganu (n = 20, 52.6%). There were 14 (36.8%) males and 24 (63.2%) females with median age of 25 years. Majority (n = 33, 86.8%) had features of thalassaemia trait with mean ± SD for Hb, mean cell volume (MCV) and mean cell haemoglobin (MCH) as 13.21 g/dL ± 1.69, 73.06 ± 4.48 fL and 24.71 ± 1.82 pg, respectively. None had evidence of haemolysis or thromboembolic complications. Six genotypes were identified; ßG-Makassar/ß,αα/αα (n = 19, 50.0%), ßG-Makassar/ßE,αα/αα (n = 4, 10.5%), ßG-Makassar/ßNewYork,αα/αα (n = 1, 2.6%), ßG-Makassar/ß,αα/-α (n = 11, 28.9%), ßG-Makassar/ß,αα/αAdanaα (n = 2, 5.3%) and ßG-Makassar/ß,αα/-SEA (n = 1, 2.6%). The ßG-Makassar/ß,αα/αα showed that features of thalassaemia trait with mean ± SD for Hb, MCV and MCH were 13.74 g/dL ± 2.40, 76.18 ± 6.02 fL and 25.79 ± 2.41 pg, respectively. This is the largest study reporting a significant number of Hb G-Makassar in Malaysia. Although the mutation is similar to Hb S, the phenotype is benign.
Fulltext Acute lymphoblastic leukemia with clonal evolution due to delay in chemotherapy: A report of a case

Haidary AM, Saadaat R, Abdul-Ghafar J, Rahmani S, Noor S, Noor S, et al.

EJHaem, 2022 Aug;3(3):1013-1017.
PMID: 36051042 DOI: 10.1002/jha2.483

Clonal evolution in acute leukemias is one of the most important factors that leads to therapeutic failure and disease relapse. Delay in therapeutic intervention is one of the reasons that leads toward clonal evolution. In this report, we present a case of acute lymphoblastic leukemia in which therapeutic delay resulted in clonal evolution that was detected by conventional karyotyping and was responsible for non-responsiveness of the disease to conventional chemotherapy. A 17-year-old boy presented with generalized body aches, rapidly progressive pallor and lethargy. Bone marrow analysis was consistent with the diagnosis of B-cell ALL. Karyotypic analysis revealed 46, XY male karyotype. The patient left the hospital due to financial reasons and after 40 days came back to the hospital. Repeated bone marrow analysis including cytogenetic studies revealed presence of three different clones of blast cells: one clone showed 46, XY with del(9p) and t (11;14), second clone showed 46, XY with del(7q) and del(9p), and the third clone showed 46, XY normal karyotype. The patient did not respond to chemotherapy and died within 1 week of induction chemotherapy (HyperCVAD-A). Timely diagnosis and institution of chemotherapy in acute leukemias patients is the key to prevent clonal evolution and thus resistance of the disease to therapeutic interventions.
Fulltext EPYSQLI (SB12; Biosimilar to Reference Eculizumab) in Asian and Non-Asian Patients With Paroxysmal Nocturnal Hemoglobinuria: Subgroup Analysis of a Global Phase III Randomized Controlled Trial

Jang JH, Tomuleasa C, Oliynyk H, Lanamtieng T, Park J, Kim Y, et al.

EJHaem, 2025 Apr;6(2):e70020.
PMID: 40123793 DOI: 10.1002/jha2.70020

INTRODUCTION: SB12 demonstrated equivalence to reference eculizumab (ECU) in complement inhibitor-naïve patients with paroxysmal nocturnal hemoglobinuria (PNH) in the previous randomized, double-blind, multi-national, crossover, Phase III study.
METHODS: The scope of this post-hoc study was subgroup analysis by race to compare the efficacy and safety of SB12 and ECU in PNH patients in the Asian and Non-Asian subgroups of the Phase III study.
RESULTS: Results including lactate dehydrogenase (LDH), number of units of packed red blood cells and safety as primary and secondary endpoints demonstrated comparable efficacy and safety of SB12 and ECU in Asian and Non-Asian PNH patients, in line with the study results in the overall population. In addition, transfusion avoidance (68.1% for SB12 vs. 72.9% for ECU, p-value of 0.4492) and hemoglobin stabilization (SB12-ECU: 6.3%, 95% confidence interval [CI] [-21.5, 34.1] and SB12-ECU: 2.5%, 95% CI [-24.8, 29.8] using stringent criteria) as post-hoc endpoints were not substantially different between SB12 and ECU treatment groups in the overall population as well as in Asians and Non-Asians.
CONCLUSION: In conclusion, this subgroup analysis by race (Asians and Non-Asians) supports comparable efficacy and safety between SB12 and reference eculizumab in global PNH patients including no difference in transfusion avoidance effect.