To determine the prevalence and geographic distribution of thalassemia and to evaluate the success of the thalassemia prevention and treatment programs in Iran. Data were obtained from the National Thalassemia Registry of Iran, Iranian Blood Transfusion Organization, genetic laboratories involved in prenatal diagnosis, related pharmaceutical companies, and centers performing bone marrow transplantation for thalassemic patients. A total of 13,879 living patients have been registered, mostly from the northern and southern parts of Iran with the median age of 15 years. Twenty-three percent of patients were older than 20 years. The number of newly diagnosed cases has been decreased considerably after the start of the prevention program. Since the introduction of prenatal diagnosis, 2819 couples (2549 fetuses) have been tested, with only 6 false results. Elective abortion was not performed in 10 affected fetuses. Most common mutations detected were IVS II-1 and IVS I-5. In 2003, approximately 25% of the national blood products and 6 million vials of desferal were used for thalassemic patients. Overall, 340 patients have received allogeneic bone marrow transplantation, of those 46 patients deceased. Bloodborne infections have also been decreased significantly owing to the national screening of blood products for bloodborne viral infections. Owing to the national prevention program and provided special care, the age distribution of thalassemic patients in Iran is getting adapted to a full prevention and treatment program and life expectancy of these patients has been increased considerably. This shift in the age distribution of thalassemia, a traditionally considered pediatric disease, will face us with new challenges and the health care system should be prepared for this new face of thalassemia.

An earthquake measuring 6.5 on the Richter scale devastated Bam, Iran on the morning of 26 December 2003. Due to the great health demands and collapse of health facilities, international aid could have been a great resource in the area. Despite sufficient amounts and types of resources provided by international teams, the efficacy of international assistance was not supported in Bam, as has been experienced in similar events in other countries. Based on the observations in the region and collecting and analyzing documents about the disaster, this manuscript provides an overview of the medical needs during the disaster and describes the international medical response. The lessons learned include: (1) necessity of developing a national search and rescue strategy; (2) designing an alarm system; (3) establishing an international incident command system; (4) increasing the efficacy of the arrival and implementation of a foreign field hospital; and (5) developing a flowchart for deploying international assistance.

Purpose: Concerns about radiotherapy-related neurocognitive sequelae in young children have led to deferral or avoidance of radiation in contemporary treatment for this fragile group of patients. We compared survival and neurocognitive outcome in two groups of infants with medulloblastoma who received adjuvant conventional craniospinal irradiation (csi) or reduced or no radiotherapy during an era of change in the philosophy of infant medulloblastoma treatment. Patients and Methods: From 1985 to 2007, 29 patients 3 years of age or younger were diagnosed and treated with curative intent in our institution. Children treated before 1994 received adjuvant radiation with chemotherapy; subsequently, radiation was prescribed essentially for disease progression or relapse. Results: Median age at diagnosis was 24 months (range: 1–36 months); 15 patients (52%) presented with metastatic disease at diagnosis. As part of initial treatment, 8 children received adjuvant radiotherapy with chemotherapy, and 21 children received postoperative chemotherapy only. Five children treated with chemotherapy alone are in prolonged remission. The 5-year event-free and overall survivals were 35.9% ± 9.8% and 50.2% ± 9.6% respectively. Extent of resection, metastatic status, and desmoplastic histology were not found to be significant prognostic factors. On serial neurocognitive evaluations, patients treated with chemotherapy with or without reduced radiotherapy demonstrated improvement of intellectual function over time. Patients treated with conventional csi exhibited significantly lower intelligence quotient scores and academic performance, with the exception of receptive vocabulary. Conclusions: Avoidance of conventional csi in treatment of very young children with medulloblastoma appears to be associated with a preserved neurocognitive profile. Neurocognitive evaluation should be integrated into the primary objectives of future infant protocols.

Hemoglobin Bart’s hydrops fetalis syndrome (BHFS) represents the most severe form of α-thalassemia, arising from deletion of the duplicated α-globin genes from both alleles. The absence of α-globin leads to the formation of nonfunctional hemoglobin (Hb) Bart’s (γ4) or HbH (β4) resulting in severe anemia, tissue hypoxia, and, in some cases, variable congenital or neurocognitive abnormalities. BHFS is the most common cause of hydrops fetalis in Southeast Asia; however, owing to global migration, the burden of this condition is increasing worldwide. With the availability of intensive perinatal care and intrauterine transfusions, an increasing number of patients survive with this condition. The current approach to long-term management of survivors involves regular blood transfusions and iron chelation, a task made challenging by the need for intensified transfusions to suppress the production of nonfunctional HbH–containing erythrocytes. Although our knowledge of outcomes of this condition is evolving, it seems, in comparison to individuals with transfusion-dependent β-thalassemia, those with BHFS may face an elevated risk of complications arising from chronic anemia and hypoxia, ongoing hemolysis, iron overload, and from their respective treatments. Although stem cell transplantation remains a viable option for a select few, it is not without potential side effects. Looking ahead, potential advancements in the form of genetic engineering and innovative therapeutic approaches, such as the reactivation of embryonic α-like globin gene expression, hold promise for furthering the treatment of this condition. Prevention remains a crucial aspect of care, particularly in areas with high prevalence or limited resources.