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Fulltext Thalassaemia Care in Terengganu – A Local Report

Nazzlin Dizana Din

Malaysian Journal of Paediatrics and Child Health, 2020;26(2):1-5.
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Inherited thalassaemia disease is commonly found in many countries of the world. Care of the disease requires comprehensive management strategies comprising of clinical management of both transfusion dependant thalassaemia (TDT) and non-transfusion dependant thalassaemia (NTDT). It also includes preventive measures such as screening programmes and genetic counseling in order to contain the genetic transmission. At the moment, the only cure is through haematopoeitic stem cell transplant (HSCT). This report illustrates thalassaemia disease prevalence in the Terengganu state and the evolution of care since National Thalassaemia Programme was launched in 2009.

Matched MeSH terms: Hematopoietic Stem Cell Transplantation
Hematopoietic Stem Cell Transplantation in T Cell and Natural Killer Cell Lymphomas: Update on Recent Advances

Boo YL, Koh LP

Transplant Cell Ther, 2021 07;27(7):571-588.
PMID: 33857661 DOI: 10.1016/j.jtct.2021.04.002

Mature T and natural killer (NK) cell non-Hodgkin lymphoma (T-NHL) has a poor prognosis. Data from existing retrospective and prospective studies have suggested that high-dose chemotherapy followed by autologous hematopoietic cell transplantation (auto-HCT) may improve the survival in patients with chemosensitive disease, either in the upfront or salvage setting. Auto-HCT is currently recommended to be used as frontline consolidation in peripheral T cell lymphoma not otherwise specified, angioimmunoblastic T cell lymphoma, anaplastic large cell lymphoma-anaplastic lymphoma kinase negative, NK/T cell (disseminated), and enteropathy-associated T cell lymphoma. However, about one-third of patients never reach transplantation because of early relapse or refractory disease. Allogeneic hematopoietic cell transplantation (allo-HCT), via its immunologic graft-versus-lymphoma effect, has been used to salvage patients with relapsed or refractory disease, resulting in long-term disease-free survival in a fraction of patients. However, the higher risk of transplant-related mortality due to regimen-related toxicities, graft-versus-host disease, and post-transplant infectious complications continues to limit the mainstream adoption of allo-HCT for this disease. Despite that, allo-HCT has been incorporated as part of the frontline treatment for aggressive subtypes of T-NHL, such as γδ T cell lymphoma and aggressive NK cell leukemia. Recent attempts to incorporate novel targeted T cell directed therapies into the treatment pathway of T-NHL may enhance treatment response and enable more patients to reach transplant, offering an alternative means of treating this disease.

Matched MeSH terms: Hematopoietic Stem Cell Transplantation*
Fulltext Survival outcomes of children with relapsed or refractory myeloid leukemia associated with Down syndrome

Raghuram N, Hasegawa D, Nakashima K, Rahman S, Antoniou E, Skajaa T, et al.

Blood Adv, 2023 Nov 14;7(21):6532-6539.
PMID: 36735769 DOI: 10.1182/bloodadvances.2022009381

Children with Down syndrome (DS) are at a significantly higher risk of developing acute myeloid leukemia, also termed myeloid leukemia associated with DS (ML-DS). In contrast to the highly favorable prognosis of primary ML-DS, the limited data that are available for children who relapse or who have refractory ML-DS (r/r ML-DS) suggest a dismal prognosis. There are few clinical trials and no standardized treatment approach for this population. We conducted a retrospective analysis of international study groups and pediatric oncology centers and identified 62 patients who received treatment with curative intent for r/r ML-DS between year 2000 to 2021. Median time from diagnosis to relapse was 6.8 (range, 1.1-45.5) months. Three-year event-free survival (EFS) and overall survival (OS) were 20.9 ± 5.3% and 22.1 ± 5.4%, respectively. Survival was associated with receipt of hematopoietic stem cell transplantation (HSCT) (hazard ratio [HR], 0.28), duration of first complete remission (CR1) (HR, 0.31 for > 12 months) and attainment of remission after relapse (HR, 4.03). Patients who achieved complete remission (CR) before HSCT, had an improved OS and EFS of 56.0 ± 11.8% and 50.5 ± 11.9%, respectively compared to those who underwent HSCT without CR (3-year OS and EFS of 10.0 ± 9.5%). Treatment failure after HSCT was predominantly because of disease recurrence (52%) followed by treatment-related mortality (10%). The prognosis of r/r ML-DS remains dismal even in the current treatment period and serve as a reference point for current prognostication and future interventional studies. Clinical trials aimed at improving the survival of patients with r/r ML-DS are needed.

Matched MeSH terms: Hematopoietic Stem Cell Transplantation*
Epidemiology of combined immunodeficiencies affecting cellular and humoral immunity- a multicentric retrospective cohort study from the Arabian Peninsula

Al-Herz W, Ziyab AH, Adeli M, Al Farsi T, Al-Hammadi S, Al Kuwaiti AA, et al.

Clin Immunol, 2023 Sep;254:109696.
PMID: 37481010 DOI: 10.1016/j.clim.2023.109696

AIMS: To understand the characteristics of combined immunodeficiency disorders that affect cellular and humoral immunity (CID) in the Arabian Peninsula.
METHODS: Retrospective study of 236 patients with CID from the region were enrolled from 2004 to 2022.
RESULTS: 236 patients were included with a majority being profound CID. Among patients with a family history of CID, the ages at onset and diagnosis, and the delay in diagnosis were lower compared to those with no family history of CID, but this did not affect time to transplant. HSCT was performed for 51.27% of the patients with median time from diagnosis to HSCT of 6.36 months. On multivariate analysis, patients who underwent early transplant had increased odds of having CD3 count ≤1000 cell/μl, diagnosed by screening or erythroderma.
CONCLUSION: There is a delay in diagnosis and treatment of CID in our region. Establishing newborn screening programs and HSCT units in our region are the urgent need.

Matched MeSH terms: Hematopoietic Stem Cell Transplantation*
Fulltext Preclinical assessments of safety and tumorigenicity of very high doses of allogeneic human umbilical cord mesenchymal stem cells

Chin SP, Saffery NS, Then KY, Cheong SK

In Vitro Cell Dev Biol Anim, 2024 Mar;60(3):307-319.
PMID: 38421574 DOI: 10.1007/s11626-024-00852-z

Human umbilical cord-mesenchymal stem cells (hUC-MSCs) have been widely investigated as a new therapeutic agent to treat injuries and inflammatory-mediated and autoimmune diseases. Previous studies have reported on the safety of low-dose infusion of hUC-MSCs, but information on the cell behaviour at higher doses and frequency of injection of the cells remains uncertain. The aim of the present study was to demonstrate the safety and efficacy of hUC-MSCs by Cytopeutics® (Selangor, Malaysia) from low to an extremely high dose in different monitoring periods in healthy BALB/c mice as well as assessing the tumorigenicity of the cells in B-NDG SCID immunocompromised mice. Umbilical cord from two healthy human newborns was obtained and the isolation of the hUC-MSCs was performed based on previous established method. Assessment of the cells at different doses of single or multiple administrations was performed on healthy BALB/c mice in dose range finding, sub-acute (7 d and 28 d) and sub-chronic periods (90 d). Tumorigenicity potential of Cytopeutics® hUC-MSCs was also evaluated on B-NDG immunocompromised mice for 26 wk. Single or multiple administrations of Cytopeutics® hUC-MSCs up to 40 × 106 cells per kilogramme of body weight (kg BW) were found to have no adverse effect in terms of clinical symptoms, haematology and other laboratory parameters, and histology examination in healthy BALB/c mice. hUC-MSCs were also found to reduce pro-inflammatory cytokines (IL-6 and TNF-α) in a dose-dependent manner. No sign of tumor formation was observed in B-NDG mice in the 26-wk tumorigenicity assessment. Single or multiple administration of allogenic Cytopeutics® hUC-MSCs was safe even at very high doses, is non-tumorigenic and did not cause adverse effects in mice throughout the evaluation periods. In addition, Cytopeutics® hUC-MSCs exhibited immunomodulatory effect in a dose-dependent manner.

Matched MeSH terms: Hematopoietic Stem Cell Transplantation*