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Objectives: The People's Democratic Republic of Laos belongs to the minority of countries worldwide without an established newborn bloodspot screening (NBS) system., Methods: In 2008, we initiated a pilot project of a neonatal screening system in the delivery suites of the Laotian capital, Vientiane. Samples were analysed for thyrotropin-stimulating hormone and 17-hydroxyprogesterone., Results: Altogether 11 362 samples were taken; an initially high recall rate dropped eventually to just above 4%. Two cases of hypothyroidism and one case of congenital adrenal hyperplasia were identified and received timely treatment., Conclusions: In summary, we have demonstrated the feasibility of establishing an NBS system in a low-resource setting as prevalent in Laos. Obstacles for the establishment of a general NBS covering the whole country include the question of financial cover, treatment costs, and adequate teaching and supervision of technicians and doctors.

Objective: To evaluate newborn screening (NBS) for very long-chain acyl-CoA dehydrogenase deficiency (VLCADD), we further characterized newborns with elevation of one or all C14-carnitine derivatives on NBS from a total of 90 338 newborns., Study Design: Palmitoyl-CoA oxidation was performed in lymphocytes to define very long-chain acyl-CoA dehydrogenase function. Molecular analysis followed in children with residual activities<50%. The acylcarnitine pattern on days 2 to 3 of life was evaluated thoroughly to define possible discrimination markers., Results: Forty newborns with increased C14:1-carnitine were identified (1:2500). In 2 newborns, VLCADD was confirmed with enzyme and molecular analyses (prevalence, 1:50,000). One of these newborns had normal results on a second screening. Also, the combination of absolute acylcarnitine values and acylcarnitine ratios did not allow correct identification of the newborn as a patient with VLCADD., Conclusions: Reliable diagnosis is not feasible with acylcarnitine analysis alone. Enzyme analysis in lymphocytes is a reliable and rapid method for correctly assessing all newborns with VLCADD and should be carried out in all newborns identified during the first screening, regardless of the results of a later acylcarnitine profile., (Copyright (c) 2010 Mosby, Inc. All rights reserved.)

Unlabelled: Polyethylene glycol conjugated asparaginase (PEG-ASNase) can be substituted in cases of hypersensitivity to native Escherichia coli asparaginase. We measured asparagine (asn) levels in plasma after a single dose of 2,500 IU/m(2) i.v. PEG-ASNase (Oncaspar) in consolidation treatment of ALL and compared those with data from the previous protocol COALL-05-92. This protocol was similar to COALL-06-97, except that children had been given 45,000 IU/m(2) C-ASNase instead of PEG-ASNase., Patients and Methods: Between May 2000 and December 2001 seventy-one children (38 boys, 33 girls) with newly diagnosed ALL treated according to the multicenter protocol COALL-06-97 were investigated in this study. Four hundred and seventy-four plasma samples (71 patients) were analysed by ion exchange chromatography after column derivatization with o-phthaldialdehyde. For comparison data (350 plasma samples) from 51 patients treated according to the protocol COALL-05-92 were available. The same method for detection of asn in plasma was used., Results: The median asparagine level in plasma after 2,500 IU/m(2) PEG-ASNase i.v. was below the limit of detection for at least 5 weeks in 81 % of the patients. When divided into high risk (HR) and low risk (LR) group, HR patients who had previously received one dose more of C-ASNase showed a markedly shorter depletion than the LR patients compatible with a higher risk of antibody formation and consequent silent inactivation after a higher number of exposures to ASNase. In the previous protocol COALL-05-92 median asn levels in plasma after 45,000 IU/m(2) native C-ASNase i.v. were below the limit of detection for at least 5 weeks in 65 % of the patients., Conclusions: 2,500 IU/m(2) PEG-ASNase led to an equally long depletion of asn in plasma as did 45,000 IU/m(2) native C-ASNase i.v. used in COALL-05-92.