ObjectiveThalassaemia is the most common inherited blood disorder in Malaysia. This study aims to report the current status of thalassaemia in Malaysia and provide a comprehensive understanding of the disease through data obtained from the Malaysian Thalassaemia Registry.DesignData were extracted from the Malaysian Thalassaemia Registry, a web-based system accessible to enrolled users through www.mytalasemia.net.my.SettingThe Malaysian Thalassaemia Registry data was recorded from reports obtained from 110 participating government and university hospitals in Malaysia.ParticipantsThe patients were those attending the 110 participating hospitals for thalassaemia treatment.InterventionData were collected from the Malaysian Thalassaemia Registry from 2007 until the fourth quarter of 2018.Primary outcome measure7984 out of 8681 patients with thalassaemia registered in the Malaysian Thalassaemia Registry were reported alive.ResultsMajority of the patients were reported in the state of Sabah (22.72%); the largest age group affected was 5.0–24.9 years old (64.45%); the largest ethnic group involved was Malay (63.95%); and the major diagnosis was haemoglobin E/β-thalassaemia (34.37%). From the 7984 patients, 56.73% were on regular blood transfusions and 61.72% were on chelation therapy. A small fraction (14.23%) has undergone splenectomy, while the percentage of patients with severe iron overload (serum ferritin ≥5000 µg/L) reduced over time. However, cardiac complications are still the main cause of death in patients with thalassaemia.ConclusionData gathered into the registry can be used to understand the progression of the disorder, to monitor iron overload management and to improve the outcomes of treatment, to enhance preventive strategies, reduce healthcare burden and improve the quality of life. Sustainability of the Malaysian Thalassaemia Registry is important for surveillance of thalassaemia management in the country and help the national health authorities to develop more effective policies.
The awareness of primary immunodeficiency (PID) in Malaysia is still not forthcoming. Certain practical issues such as lack of clinical immunologists and specialized laboratory diagnostic facilities remain to be addressed. However, great efforts taken by passionate clinicians and scientists in the immunology networking have ascertained some prevalence. Despite the limitation, all suspected cases of PID are being properly investigated and competently managed. In this case report we highlighted the obstacles we faced in managing PID patients, particularly preparing for bone marrow transplant. This is the first transplanted case of chronic granulomatous disease in Malaysia, which emphasizes the importance of collaborative work to ensure further morbidities or mortalities are prevented.
INTRODUCTION Patients with high risk medulloblastoma are treated either with high dose chemotherapy or hyperfractionated radiotherapy. Both approaches are not feasible in resource-limited countries. POG9031 trial has reported favourable outcome for high risk medulloblastoma using standard chemotherapy and radiotherapy only. Hence, we have adopted the protocol using chemotherapy first approach due to logistical reasons. OBJECTIVE To review the outcome of children diagnosed with high risk medulloblastoma in Hospital Kuala Lumpur. METHODS Patients diagnosed with high risk medulloblastoma between January 2015 and June 2018 treated using the chemotherapy first approach as per POG9031 protocol were identified. Data was then extracted and analysed. RESULTS Nine patients were identified, 3 boys and 9 girls. Median age was 9.3 years (range 2.6 – 15.9 years). Median follow up for survivors are 3.6 years. Five patients (55.6%) had macroscopic metastatic disease at diagnosis. All patients had significant residual disease post-op. Only 3 patients are disease free till last follow up, giving a 3 years event free survival of 16%. Of the 6 patients who had relapsed, 4 have died, giving a 3 years overall survival of 46%. Patients with no metastasis at diagnosis (M0) fared better with 3 years event free survival of 38%, but 3 years event free survival for patients with macroscopic metastatic disease (M+) was 0%. CONCLUSION Outcome of children with high risk medulloblastoma treated with chemotherapy first approach was dismal.
BACKGROUND: Radiation treatment of GCT brain, in particular germinomas which are highly radio-sensitive,over the years has evolved from the cranio-spinal irradiation(CSI) to more conformal treatment like whole ventricle radiation followed by tumor bed boost (WV + TB), without compromising the results. We report our experience of treatment outcome of patients treated consecutively over a period of more than ten years with both the methods. METHOD: Twelve consecutive patients registered and treated in radiotherapy department between 2000 and 2013 after surgery (biopsy/decompression) and chemotherapy, were analysed in December 2013. Total Number (Histo-pathology) 12 (Germinoma-9, Non-Germinoma-2, No histology-1); Median age (Years) 13 (Range 5-24 years); Gender-M/F (%) 10:2(83:17); Co-morbidities None; Pre-Surgery KPS .90 (92%); Surgery (n) Biopsy/Decompression+ VP shunt (11), No Surgery (1); Sub-site Pineal-7(58%), Supra-sellar-5(42%). TREATMENT: Ten patients received 3D-CRT, while two received IMRT. Three received CSI while nine received WV + TB. CSI dose ranged from 23.4 Gy -36 Gy in 13-20 fractions. For WV the intended dose was 36Gy/20 fractions followed by 9Gy/5 fractions to TB. Pre-radiation all received combination chemotherapy, cisplatin or carboplatin + etoposide + irinotecan. RESULTS: All the patients tolerated and completed treatment with no serious adverse effects. Follow-up was done with serial tumor markers, hormonal, visual, auditory assessment and MRI. Two patients,both non-germinomas treated with CSI progressed and died with-in one year of treatment. Ten patients (9-germinomas, 1-no histology) till date are doing fine,without any loco-regional or distant failures. Ten year overall survival is around 80%.Two patients have developed hormonal imbalance and are on treatment,while one of them developed progressive bilateral optic atrophy which was present prior to radiotherapy. CONCLUSION:Our results are comparable with the reported literature.The overall survival in Germinomas is 100%,Treatment related effects are minimal as a result of reduced dose and limited treatment volume,-contributing possibly to better outcome along with various other reasons. BACKGROUND: Intracranial germ cell tumor (ICGCT) represents 3-15% of pediatric brain tumors. According to the current WHO classification, ICGCTs are classified as germinomas and non-germinomatous germ cell tumors. Germinomas are exquisitely radiosensitive, and chemo-radiotherapy is equally effective. Non-germinomatous germ cell tumors, though more aggressive than germinomas, can be treated effectively with intensive chemo-radiotherapy. ICGCTs in infants and very young children are typically mature teratomas which have excellent outcome after gross total resection. ICGCTs excluding mature teratomas, in this young age group, are extremely rare. There is also no published series or reported incidence of ICGCTs which excludes mature teratomas in children younger than 3 years of age. METHODS AND MATERIALS: We describe a series of 15 ICGCTs excluding mature ter...
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