Hematopoietic stem cell transplantation with reduced toxicity conditioning regimen in mitochondrial neurogastrointestinal encephalopathy syndrome

Background Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is a rare autosomal recessive disorder due to mutations in the TYMP gene. Clinical findings are characterized by neurologic manifestations and severe gastrointestinal dysfunction. The syndrome is usually fatal, the most effective treatment appears to be hematopoietic stem cell transplantation (HSCT). Procedure In this retrospective study, we evaluated HSCT that was performed using a reduced toxicity myeloablative conditioning regimen in patients with MNGIE at our center. Results A total of six allogeneic transplant procedures were performed in four patients. Three patients had fully matched donors, and one patient had a haploidentical donor. Treosulfan-based myeloablative conditioning regimen was applied in five of six transplants. Bone marrow was used as a stem cell source. One patient is being followed up in the 4th year of posttransplant with full chimeric and without graft versus host disease (GVHD). One patient died of acute stage IV gastrointestinal system GVHD. Two patients underwent second transplantation due to engraftment failure, one of which was the patient who had a haploidentical transplant. Conclusions Treosulfan-based regimen is well tolerated, although engraftment failure with this conditioning regimen can be a significant problem. We share our haploidentical transplant experience, which will be the first reported case in the literature

Aflatoxin: Is it a neglected threat for formula-fed infants?

WOS: A1997WP87100007PubMed ID: 9124050In the present study, the risk of exposure to aflatoxin in infants fed breast milk and formula was investigated. For this purpose, aflatoxin B-1 (AFB(1)) was determined in the serum of both breast-fed and formula-fed infants. Serum AFB(1) positivity was significantly higher in the formula-feeding (F) group than the breast-feeding (B) group (42.8 vs 8.5%, P < 0.01). The AFB(1) concentration in different commercial formulas was also determined. Aflatoxin B-1 was found in seven of the eight newly opened packages of different brands of formula. The concentrations showed a statistically significant increase at the 30th day after opening the packages (P < 0.01). Although AFB(1) concentrations in the formulas were found to be within acceptable limits for most countries, still, its existence must be carefully evaluated because future influences of very small amounts of aflatoxin on the growing organism have not been fully elucidated. Therefore, it was again concluded that for infants, human milk is safer than commercial formulas because of the lower contamination risk of aflatoxin. Also, commercial formulas must be regularly examined by authorities for the possible risk of aflatoxin contamination

Low-dose oral methotrexate therapy in resistant juvenile rheumatoid arthritis [Direncli juvenil romatoid artritte dusuk doz oral metotreksat tedavisi]

Twelve children with resistant juvenile rheumatoid arthritis were given 10 mg of oral methotrexate per square meter of body-surface area per week for eight to 24 months (mean±SD, 18±6 months). The patients were eight to 16 years old (mean 10.9±2.4 years) and the mean duration of the disease was 4.5±1.6 years (range, two to nine years). The patients had active polyarthritis at the onset of the methotrexate therapy. All the patients had used hydroxychloroquine, and four of them also sulfasalazine as second-line agents. Methotrexate therapy decreased swelling count by a mean of 47 percent, swelling index by 55 percent, articular count 52%, articular index by 54 percent and morning stiffness by 65 percent. In addition, methotrexate therapy increased hemoglobin value by more than 2 gm/dl in four (33%) patients and decreased the number of platelets by more than 200,000/mm3 in three (25%) patients. The erythrocyte sedimentation rate was 80±26 mm/hr and 32±18 mm/hr before and after methotrexate therapy, respectively. No adverse effects severe enough to withdraw methotrexate therapy were observed during the study. Compliance with methotrexate therapy was complete in all patients. From the data presented here, we conclude that low-dose oral methotrexate is an effective treatment for children with resistant juvenile rheumatoid arthritis. We recommend earlier consideration of methotrexate in place of other slow-acting antirheumatic drugs of cases of juvenile rheumatoid arthritis not responding well to usual therapy

Letter to the editor: Primary cardiac rhabdomyosarcoma

WOS: 000173435400022PubMed ID: 1181319