Evolution of Hematopoietic Stem Cell Transplant Programs in Malaysia.

Hematopoietic stem cell transplantation (HSCT) is now widely practiced worldwide. It has the potential to cure many hematological diseases, such as acute leukemia and thalassemia. As an emerging country, Malaysia has made advancements despite many challenges. HSCT has evolved rapidly since the first pediatric allogeneic HSCT case in 1987. The first adult HSCT was performed 5 years later in 1993. Currently, a total of 13 hospitals offer HSCT services throughout Malaysia. These include private healthcare services, substantially funded government hospitals governed by the Ministry of Health, and partially funded teaching hospitals governed by the Ministry of Education. Until 2015, 1,987 allogeneic and 1,648 autologous HSCT procedures were performed. This article narrates the history and development of HSCT in Malaysia and briefly discusses the challenging issues in this area.

The safety, tolerability, and efficacy of a liquid formulation of deferiprone in young children with transfusional iron overload.

Limited data are available on the use of deferiprone in children younger than 10 years of age. This study evaluated the safety and efficacy of a new liquid formulation of deferiprone for the treatment of transfusional iron overload in children 1-10 years old. One hundred children (91 thalassemia major, 8 Hb E-ß thalassemia, and 1 sickle cell disease) were enrolled for a 6-month treatment with deferiprone (50 to 100 mg/kg/d). The safety profile was similar to or better than that reported in earlier studies with deferiprone tablets in older children and adults. No unexpected adverse reactions were observed. Gastrointestinal intolerance (GI) was observed in 11% and an increased serum ALT in 12% of the children. Both events were transient. Mild neutropenia, observed in 6% of patients, did not progress to agranulocytosis and resolved despite continuous deferiprone treatment. Two patients experienced agranulocytosis that resolved without complications upon discontinuation of therapy. Deferiprone use was associated with a significant decline in mean serum ferritin level from 2532±1463 µg/L at baseline to 2176±1144 µg/L (P<0.0005). The results of this study show a favorable benefit/risk ratio of deferiprone oral solution for the treatment of young children with transfusional iron overload.

Cefepime monotherapy for treatment of febrile neutropenia in children.

AIM: Empirical therapy for children with febrile neutropenia has traditionally consisted of combination antibiotics, usually a beta-lactam and an aminoglycoside. However, recent trends and international guidelines have now made monotherapy a feasible option in the management of this group of patients. We prospectively evaluated the efficacy and safety of cefepime monotherapy in our population of paediatric cancer patients with febrile neutropenia. METHODS: An audit was performed on children aged 16 years and younger presenting with fever and neutropenia who were managed with empirical single-agent cefepime. The patients were analysed for clinical outcome, documented infections and side-effects of the study drug. Success was defined as clinical improvement without treatment modification. Death or any change to the empirical antibiotic was considered as failure. RESULTS: In this study 79 children (median age 5.2 years) with 133 episodes of febrile neutropenia were prospectively studied between August 2004 and August 2005. A microbiologically documented infection was seen in 26 episodes. The success rate of cefepime monotherapy was 60%. The rate of survival through neutropenia (with or without modification) was 98%. No significant adverse effects were seen. CONCLUSION: Cefepime monotherapy is a safe and feasible option for treatment of childhood cancer patients with febrile neutropenia.

Cryopreservation of mobilized blood stem cells at a higher cell concentration without the use of a programmed freezer.

Cryopreservation of peripheral blood stem cells (PBSC) mobilized by chemotherapy combined with or without granulocyte colony-stimulating factor (G-CSF) is an essential part of procedure for anti-cancer strategies. We evaluated whether a higher cell concentration (2 x 10(8)/ml) without the use of a programmed freezer was acceptable for the storage of mobilized PBSC in an autologous setting. Mobilized PBSC were enriched to mononuclear cells (MNC) by Percoll separation and then frozen at cell concentrations of 2-5 x 10(7)/ml (group I, n=20) or 2 x 10(8)/ml (group II, n=44) without the use of a programmed freezer using 5% DMSO, 6% hydroxy ethyl starch, and 4% autologous serum or human albumin. CD34+ cells purified by ISOLEX300 were frozen at 2 x 10(7)/ml (group III, n=22) using the same method. The median recovery rates of CD34+ cells and CFU-GM were, respectively, n.d. (not determined) and 88% in group I, 103 and 64% in group II, and 98 and 53% in group III. There was a statistical significance between the recovery rate of CFU-GM in group III and that in group I ( p=0.02). The median percentage of cell viability after thawing in each group was 89, 87, and 75%, respectively. The median numbers of days after PBSCT to achieve a WBC of >1.0 x 10(9)/l, an absolute neutrophil count of >0.5 x 10(9)/l, and a platelet count of >50 x 10(9)/l were, respectively, 11, 11 and 15 in group I; 12, 12 and 16 in group II; and 12, 12 and 27 in group III. These results suggest that enriched MNC from mobilized PBSC could be frozen at a higher cell concentration (2 x 10(8)/ml) without the use of a programmed freezer, leading to reduction of the toxicities associated with infusion of thawed cells and of costly space required for cell storage.