Italian consensus statement on paediatric HIV infection.

The objective of this document is to identify and reinforce current recommendations concerning the management of HIV infection in infants and children in the context of good resource availability. All recommendations were graded according to the strength and quality of the evidence and were voted on by the 57 participants attending the first Italian Consensus on Paediatric HIV, held in Siracusa in 2008. Paediatricians and HIV/AIDS care specialists were requested to agree on different statements summarizing key issues in the management of paediatric HIV. The comprehensive approach on preventing mother-to-child transmission (PMTCT) has clearly reduced the number of children acquiring the infection in Italy. Although further reduction of MTCT should be attempted, efforts to personalize intervention to specific cases are now required in order to optimise the treatment and care of HIV-infected children. The prompt initiation of treatment and careful selection of first-line regimen, taking into consideration potency and tolerance, remain central. In addition, opportunistic infection prevention, adherence to treatment, and long-term psychosocial consequences are becoming increasingly relevant in the era of effective antiretroviral combination therapies (ART). The increasing proportion of infected children achieving adulthood highlights the need for multidisciplinary strategies to facilitate transition to adult care and maintain strategies specific to perinatally acquired HIV infection.

[Cyanosis in adult with congenital heart disease]. / Trattamento del paziente cianotico.

Cyanosis is negative predictor of survival in adult patients with congenital heart disease. When cyanosis is secondary to heart or lung disease, chronic hypoxiemia result in hematologic, neurologic, renal and reumatic complications . Is important,for the optimization of therapeutics procedure, the follow up of heart disease and of oxygen saturation and blood work. For the care of this patients are required specialized centers with a multidisciplinary team of experts.

Hormonal, auxological and clinical follow-up in children with connatal HIV infection. Personal records.

AIM: HIV infection and antiretroviral drugs have relevant endocrine implications, affecting growth and pubertal development. Moreover stature impairment cannot depend only on decreased hormonal secretion. METHODS: We studied for 7 years growth, puberty, bone maturation, hormonal secretion [Growth Hormone (GH) basal and after stimulation with Clonidin and Insulin, Insulin-like Growth Factor 1 (IGF-1), Insulin-like Growth Factor Binding Protein 3 (IGFBP-3), FSH, LH- gonadic hormones axis, ACTH, Cortisol, TSH, fT4, T4, T3, anti-thyroid antibodies, Leptin] of 10 HIV-infected children. RESULTS: In 3 patients stature was <-2 SDS in the first 2 years and in prepubertal age, with intervals of improved growth. The weight was >2 SDS in 6 children, <-2 SDS in 1 girl, while the other 3 patients had a weight <-2SDS only in the first 2 years of life. Height growth velocity was >10 degrees Centile all over the years of follow-up in 9 patients, while weight growth velocity was pathological in 5. Leptinemia showed higher levels at the beginning of follow up: 0.82-11.68 ng/L (M+/-DS: 3.29+/-4.15) than at the end of the study: 0.2-3 ng/L (M+/-DS: 1.65+/-1.01). Leptin levels showed a statistically significant correlation with CD4/CD8 count (P: 0.010; r: 0.916) and with the CDC stage (P: 0.006; r: 0.937), meaning a strong link to the severity of the disease. CONCLUSIONS: A good clinical control of HIV infection can guarantee growth within physiological centile in most of HIV-infected children. Over all IGFBP-3 and IGF-1 are good markers of growth, more usable than GH.

Exercise capacity in apparently healthy survivors of cancer.

AIMS: To evaluate cardiopulmonary exercise tolerance in a large cohort of apparently healthy paediatric cancer survivors in order to determine their participation in sporting activities. METHODS: A total of 84 young (<21 years) asymptomatic childhood cancer survivors, who had been exposed to anthracyclines (mean dose 212 mg/m2) and/or chest irradiation (median dose 2000 cGy), with normal left ventricular systolic function at rest (fractional shortening >29%), and 79 healthy controls were studied. Exercise testing was performed on a treadmill ergometer. Gas exchange analysis and derived variables were measured on a breath-by-breath basis. Pulmonary functional evaluation was performed before exercise. Echocardiographic evaluation at rest was performed within one month before the exercise test. RESULTS: There were no differences in exercise responses between patients and controls. In boys <13 years, mean VO2 max was slightly but significantly lower than in controls. This finding was thought to be a result of decreased physical fitness as all the other exercise parameters were similar to those in the controls. CONCLUSIONS: Results show that apparently healthy survivors of paediatric cancer can take part in dynamic sporting activities if they exhibit a normal response to cardiopulmonary exercise testing, while those that exhibit a reduced VO2 max should be re-evaluated after an aerobic training programme, and should undergo tailored dynamic physical activity if the VO2 max does not normalise.

Production process contamination of citrus essential oils by plastic materials.

Twelve samples of plastic materials employed in the industrial extraction of citrus essential oils were analyzed for the presence of phosphorated plasticizers, chloroparaffins, and phthalate esters. The samples tested were found to release these contaminants into the essential oils during the production process. Contamination tests were carried out using dichloromethane and uncontaminated samples of citrus essential oils as extractants. The extracts were analyzed by HRGC/FPD, ECD, and MS. Only one sample of plastic material was found to release triaryl phosphates, 5 samples released chloroparaffins, 6 released diisobutyl phthalate, and 8 released bis(2-ethylhexyl) phthalate. Significantly larger quantities of contaminants were released by new plastic parts than by used plastic parts.

Contamination of Italian citrus essential oils: presence of chloroparaffin.

In this study, the contamination by chloroparaffin of Sicilian and Calabrian citrus essential oils, produced in the crop years 1994-1996, was investigated. The analyses were carried out on 102 lemon oils, 98 orange oils, and 96 mandarin oils, using a dual-channel GC-ECD. It was found that 53% of lemon oil, 33% of orange oil, and 38% of mandarin oil samples were contaminated. The mean contamination levels were 7.1 ppm (lemon), 2.5 ppm (orange), and 5.3 ppm (mandarin). The highest concentration of chloroparaffin found was 60 ppm in a lemon oil sample.

Organochlorine pesticide residues in Italian citrus essential oils, 1991-1996.

Organochlorine pesticide contamination in 148 lemon essential oils, 123 sweet orange oils, 121 mandarin oils, and 147 bergamot oils produced in Italy in the years 1991-1996 was studied by HRGC-ECD. Confirmation analyses were carried out by GC-MS. Tetradifon, dicofol and its decomposition product 4,4'-dichlorobenzophenone were found. Over the course of the study dicofol and tetradifon residues steadily decreased; the percentage of contaminated samples reflects this course and decreases considerably from 1991 to 1996.

Effects of fluvastatin treatment on red blood cell Na+ transport systems in hypercholesterolemic subjects.

This study was performed to ascertain the effects of short-term cholesterol-lowering therapy with fluvastatin on red blood cells Na+ transport systems. Forty familial hypercholesterolemic subjects (FH; 19 men and 21 women) without hypertension or cardiovascular disease were given a placebo for 4 weeks, and then randomized in two groups. Twenty (fluvastatin group) were given fluvastatin (40 mg/day), and the other 20 (placebo group) continued placebo administration. After the placebo period and after 4 and 12 weeks of placebo or fluvastatin treatment, we measured Na+/K+ pump activity, Na+/K+ cotransport (Na+/K+ Ct), Na+/Li+ countertransport (Na+/Li+ Cnt), passive Na+ permeability (Na+PP), and internal Na+ content (Na+i). The same parameters were measured in 23 control subjects (C) with normal cholesterolemic values, who were matched for sex and age. FH had higher Na+/Li+ Cnt values than C (193.2 +/- 59.4 vs. 139.8 +/- 48.7 microM cells/h; p < 0.01), an increase in Na(+)PP (0.034 +/- 0.012/h vs. 0.018 +/- 0.004/h; p < 0.001), and higher Na(+)i (7.5 +/- 1.5 vs. 6.2 +/- 0.9 mM cells; p < 0.001). In hypercholesterolemic subjects, Na(+)i values were correlated with cholesterol (total and LDL) and apo B levels, whereas an inverse correlation was found for HDL-c and apo AI levels. Reduced total and LDL cholesterol and apo B levels after fluvastatin treatment caused a decrease in both Na(+)/Li(+) Cnt (from 186.1 +/- 60.5 to 125.1 +/- 34.0 microM cells/h; p < 0.001) and Na(+) PP (from 0.035 +/- 0.013/h to 0.02 +/- 0.016/h; p < 0.01), and an increase in Na+/K+ pump activity (from 1,549.0 +/- 507.7 to 1,894.2 +/- 536.2 microM cells/h; p < 0.04), with a significant reduction in the internal Na+ content (from 7.5 +/- 1.6 to 5.8 +/- 2.4 mM cells; p < 0.001). Our findings show that hypercholesterolemia affects red blood cell Na+ transport systems, with an increase in Na+/Li+Cnt, Na+PP, and the internal Na+ content. Cholesterol-lowering treatment with fluvastatin influences Na+ transport systems and reduces the internal Na+ content. This might also be responsible for the greater vascular reactivity observed in hypercholesterolemic patients, and its amelioration after a reduction in cholesterol levels.

Limited value of zinc protoporphyrin as a marker of iron status in chronic hemodialysis patients.

BACKGROUND: In an attempt to find new parameters able to evaluate the actual iron availability by bone marrow cells, zinc protoporphyrin (ZnPP), a metabolic intermediate generated in the red blood cell by the incorporation of zinc instead of iron, has been proposed. ZnPP is a good marker of iron-deficiency anemia in non-uremic people, as red blood cell ZnPP concentration rises specifically (except for lead intoxication) in this condition. Existing data on ZnPP as a marker of iron deficiency in uremic patients comes mainly from cross sectional studies on chronic hemodialysis and has produced conflicting results. SUBJECTS AND METHODS: Therefore, we prospectively studied 42 HID patients, 28-88 years old, 13-346 months of dialysis age, beginning from a period of maximal iron deficiency, due to the lack of parenteral iron compounds (T0) up to the end of more than one year of follow-up with continuous parenteral iron supplementation (T4). ZnPP, hemoglobin, transferrin saturation and ferritin were serially determined before and after six weeks (T1), four months (T2), seven months (T3) and 14 months (T4) of parenteral iron supplementation at a maintenance dose of 0.5-1 mg/kg/week. RESULTS: In comparison with baseline values (95+/-37 micromol/mol heme) there were no significant changes in ZnPP levels at T1 and T2 despite a continuous increase in both transferrin saturation and ferritin values, while ZnPP significantly decreased at T4 (63+/-37 micromol/mol heme, p<0.001). There was no correlation between ZnPP and both transferrin saturation and ferritin at any time during the study, the same was true for ZnPP and zinc and lead serum concentration, fibrinogen and reactive C protein levels at T1 and T4, respectively. At T4, only 2/10 patients who still showed ZnPP levels >80 micromol/mol heme had absolute or functional iron deficiency, when the percentage of hypochromic red cells were measured. CONCLUSION: We conclude that ZnPP untimely parallels a change in iron balance in only a proportion of uremic people, in as much as confounding factors, such as chronic inflammation and uremia in itself may obscure its relationship with iron status. Therefore, ZnPP cannot be assumed to be a first-line diagnostic marker of iron balance in uremic patients.

Contamination of Italian citrus essential oils: presence of phthalate esters.

Contamination by phthalate esters of Sicilian and Calabrian citrus essential oils, produced in the crop years 1994-1996, was investigated using a GC-MS system with direct injection of the samples. A total of 35 lemon oils, 31 orange oils, and 21 mandarin oils were analyzed. Diisobutyl phthalate and/or bis(2-ethylhexyl) phthalate were found in almost all samples, while di-n-butyl phthalate was present in 8. Concentrations up to a maximum of 62 ppm were found for diisobutyl phthalate and up to a maximum of 29.9 ppm for bis(2-ethylhexyl) phthalate.

[Lymphedema of the upper extremity in patients operated for carcinoma of the breast: clinical experience with coumarinic extract from Melilotus officinalis]. / Linfedema dell'arto superiore in pazienti operati per carcinoma della mammella: esperienza clinica con estratto cumarinico di Melilotus officinalis.

OBJECTIVES: The aim of this clinical study was to verify the therapeutic activity of cumarinic extract of Melilotus officinalis (CEMO) in patients with chronic lymphedema of the upper arm caused by lymphadenectomy for breast cancer. Cumarine, in fact, has antiedemic properties due to macrophagic action that stimulates proteolysis in the tissues affected by chronic lymphedema. PATIENTS AND METHODS: In an open clinical study we enrolled 24 patients with chronic upper arm lymphedema due to post-lymphadenectomy of the axilla for breast cancer. 21 patients were eligible to receive 400 mg of CEMO containing 8 mg of cumarine in a sole daily administration for 6 months. We measure the circumference of the upper arm at 3 and 6 months from treatment. We evaluated the symptoms and tolerability through a questionnaire given to the patients at every clinical control. RESULTS: Of the 21 (87.5%) patients eligible, only 14 (66.6%) were treated with CEMO according to protocol. Of these 11 patients (52.3%) had a reduction of the circumference of the affected arm of 5% with respect to base values. Three patients (14.2%) had no change. In 12 patients (57.1%) symptoms improved. As for tolerability: 3 patients (14.2%) had transitory gastrointestinal side-effects. There was worsening of lymphedema and symptoms in 4 patients (19%) that did not receive CEMO and were followed as controls. Three patients (14.2%) were not evaluable because they were lost to follow-up. CONCLUSIONS: Cumarinic extract of Melilotus officinalis (CEMO) was effective in reducing lymphedema in 79% of the pts treated for a period of six months. The median reduction of the upper arm circumference was modest (5% with respect to initial values) but statistically significant (p = 0.048). Treatment with CEMO for lymphedema could be associated to the physiotherapy given to these patients such as manual lymph drainage (MLD).

Erythrocyte passive potassium flux is increased in patients with ischemic coronary disease (ICD) and in subjects with family history of ICD.

BACKGROUND: It has been proposed that ischemic coronary disease (ICD) associated potassium loss could be due to modifications of potassium permeability. We investigated whether a positive family history of ICD can influence this parameter. We have compared potassium permeability in erythrocytes from ICD patients and from positive family history subjects (FICD) with control subjects. METHODS: All patients and subjects were carefully selected for the absence of hypertension and dysmetabolic pathologies. ICD group: 24 patients (19 males, 5 females; ages 43 to 69) all affected by ischemic coronary disease, under no drug treatment; FICD group: 18 subjects (all males, ages 27 to 42) with a verified positive ICD family history, without hypertensive family history and cardiovascular pathology; control group: 16 subjects (11 males, 5 females; ages 28 to 48) without positive family history of ICD. Passive potassium efflux (PPE) was spectrophotometrically measured in K-free medium containing ouabain and bumetanide. The kinetic constant was calculated by dividing PPE by the erythrocyte potassium concentration. RESULTS: No statistically significant differences were noted between the intracellular potassium content of the three groups. However, (1) the passive potassium permeability of the ICD group was significantly higher (kK=0.055 +/- 0.021 h(-1), n=24) than that of the control group (kK=0.023 +/- 0.008 h(-1), n= 16; p<0.00001), (2) the FICD group was higher (kK=0.036 +/- 0.012 h(-1), n=18) than the control group (p<0.001), and (3) the ICD group was higher than the FICD group (p<0.001). CONCLUSIONS: Our results suggest an inheritability of ICD, paralleling the familial aggregation of the pathology. Erythrocyte potassium permeability could represent an early marker of ischemic coronary disease and be used as a prophylactic tool.

[Effects of adrenergic stimulation on transmembrane transport of Na+ in patients with essential hypertension]. / Effetti della stimolazione adrenergica sui sistemi di trasporto transmembrana del Na+ in pazienti con ipertensione essenziale.

BACKGROUND: It has been studied whether an adrenergic stimulation induced by the cold pressor test (CPT) could influence the behaviour of the transmembrane transport systems of sodium in hypertensive subjects compared to a normotensive control population. MATERIALS AND METHODS: Twenty-two hypertensive subjects (average age 43.2 +/- 5.7 years), with normal weight, without signs of cardiovascular and metabolic diseases, underwent the cold pressor test. The dynamic behaviour of sodium erythrocytic transport systems and plasmatic norepinephrine was evaluated basally, at the third minute during the cold pressor test and 20 minutes after the end of the test. The same test was carried out in a control population made up of 20 normotensive subjects (average age 41.9 +/- 4.8 years), selected on the basis of the absence of any cardiovascular or metabolic pathology and without family history of arterial hypertension. RESULTS: The cold pressor test did not cause significant changes in the sodium transmembrane transport systems in normotensive subjects, while in the hypertensive subjects a significant reduction was observed, during the test, in the total efflux of sodium and in the sodium/potassium pump, respectively from 2636 +/- 296 mumol/l/red blood cells/hr to 2032 +/- 178 mumol/l/red blood cells/hr (p < 0.0001) and from 2156 +/- 149 mumol/l/red blood cells/hr to 1610 +/- 101 mumol/l/red blood cells/hr (p < 0.0001); the intraerythrocytic sodium increased from 6.5 +/- 1.0 mmol/l/cells to 7.2 +/- 1.1 mmol/l/cells (p < 0.04) and the passive permeability decreased from 0.039 +/- 0.004 hr-1 to 0.018 +/- 0.006 hr-1 (p < 0.0001). During cold pressor test the increase in the plasma norepinephrine levels was correlated to the reduction in the total efflux of sodium (r = -0.60; p < 0.003) and in the sodium/potassium pump (r = -0.59; p < 0.003) only in hypertensive subjects. CONCLUSIONS: Our data show that an adrenergic stimulation, induced by the cold pressor test, is able to significantly influence the behaviour of transmembrane fluxes of sodium in hypertensive subjects, and it causes an inhibitory effect on the sodium/potassium pump and an increase in the intraerythrocytic sodium. Such data show the existence in hypertensive subjects of an interrelationship between adrenergic activity and sodium transport systems that could cooperate in causing and/or in maintaining the hypertensive syndrome.

[Approach to male breast cancer. Our experience]. / L'approccio al cancro della mammella maschile. Nostra esperienza.

Male breast cancer is a rare neoplasia and represents only 1% of all forms of breast cancer. The etiopathogenesis of breast carcinoma is still unknown in both males and females, but an altered hormonal metabolism may play a role in its genesis. The prognosis of breast cancer is slightly worse in males, especially in cases with axillary lymph node metastases. The paper discusses the therapy administered to 8 patients with male breast cancer who were treated by the authors between January 1983 and April 1990.

[Effect of beta blockade on the erythrocyte transport of Na+: evaluation during stimulation by cold pressure test in patients with essential hypertension]. / Effetti del beta-blocco sui sistemi di trasporto eritrocitario del Na+: valutazione durante stimolo con cold pressor test in pazienti con ipertensione essenziale.

Several studies have shown in essential hypertension alterations of the transmembrane red blood cells sodium fluxes, as an involvement, especially in the early phases, also of the adrenergic system. In this study we evaluated the behaviour of red blood cells fluxes of sodium before, during and after the cold pressor test, a method used also to evoke an adrenergic stimulation, in twenty hypertensive subjects, 14 males and 6 females, with an average age of 43.2 +/- 5.7 years, with normal weight and without cardiovascular complications and metabolic diseases. The behaviour of the Na+ total efflux (Na+ TE), of the Na+/K+ pump, of the Na+K+ cotransport (Na+/K+ CT), of the Na+/Li+ counter transport (Na+/Li+ Cnt), of the Na+ passive permeability (Na+ PP), of the intracellular Na+ (I Na+) and of the plasmatic noradrenaline (NE) was evaluated basally, at the third minute during cold pressor test (CPT) and 20 minutes after the end of the test. The test, which the same method, was repeated after a 30 day treatment with propranolol at the dose of 240 mg/day in three daily administrations. The beta-blockade caused, besides the reduction of both the systolic and diastolic pressure values, a significant increase in the Na+/K+ CT (from 248 +/- 41 to 314 +/- 71 mmol/l/cells/h, p < 0.001) and a decrease in the Na+ PP (from 0.039 +/- 0.004 to 0.023 +/- 0.007 hr-1, p < 0.00001), probably directed towards the reduction of the accumulation of intracellular Na+, that could compete, among the other mechanisms, with the anti-hypertensive action of the beta-blockers. The CPT caused, before the beta-blockade, a significant depression of the Na+/K+ pump (from 2057 +/- 149 to 1610 +/- 101 mmol/l/cells/h, p < 0.00001) and of the Na+ TE (from 2640 +/- 397 to 2032 +/- 179 mmol/l/cells/h, p < 0.00001) inversely correlated to the levels of NE (r = -0.60, p < 0.003), with a consequent increase in I Na+ (from 6.2 +/- 0.6 to 7.5 +/- 1.5 mmol/l/cells, p < 0.001), showing how the adrenergic activation in hypertensive subjects is able to interfere with the systems of transmembrane transport with an inhibitory attitude, that is expressed by an increase in the levels of I Na+. The beta-blockade was able to outweigh the depression of the Na+/K+ pump (from 1843 +/- 584 to 1728 +/- 640 mmol/l/cells/h, p: ns) and the reduction of the Na+ TE, preventing the accumulation of I Na+ (from 6.3 +/- 1.6 to 6.6 +/- 1.3 mmol/l/cells, p: ns). Such data show an increased susceptibility of the Na+ transport systems to the adrenergic stimuli in hypertensive subjects with a tendency to favor the accumulation of I Na+ and that the beta-blockade is able to antagonize the effects, with a maintenance of the intracellular levels of Na+.

[Transmembrane sodium transport systems in various forms of hyperlipoproteinemia]. / Sistemi di trasporto transmembrana del Na+ in forme diverse di iperlipoproteinemia.

The possible interrelationships between the erythrocytic transport systems of Na+ (Na+/K+ pump, Na+/K+ cotransport, Na+/Li+ countertransport, Na+ passive permeability) and the plasmatic lipids (cholesterol, triglycerides, HDL, LDL, apoprotein A1, apoprotein B) were studied in 42 normotensive subjects with different forms of hyperlipoproteinaemia and with a negative familiarity for arterial hypertension. In subjects with hypercholesterolaemia (hyperlipoproteinaemia II A and II B) an elevated activity of the Na+/K+ pump was noticed, while in subjects with hypertriglyceridaemia (type IV) an increase in Na+ passive permeability and Na+/Li+ countertransport with a lower level of intraerythrocytic Na+ was shown. A negative correlation was observed between the total efflux of Na+ and Na+/K+ pump and the levels of cholesterol (r = -0.43, p < 0.04 and r = -0.41, p < 0.05) and the apoprotein B/A ratio (r = 0.42, p < 0.05 and r = -0.50, p < 0.01). A negative correlation was also noticed between the Na+/K+ pump and the levels of apoprotein B (r = -0.41, p < 0.05). The Na+/K+ cotransport appeared inversely correlated with the levels of HDL cholesterol (r = -0.42, p < 0.05), while the Na+ passive permeability was negatively correlated with the levels of LDL (r = -0.43, p < 0.04) and positively correlated with the plasmatic triglycerides (r = +0.54, p < 0.01). Such data show that the plasmatic lipids can influence the systems of transmembrane ionic transport of Na+ and play an important role also this way, in cardiovascular pathology.

Effects of cloricromene on the levels of endothelin and on the microcirculatory function in peripheral atherosclerotic arteriopathies.

The effects of cloricromene on plasma endothelin-1 (ET-1) levels and on microcirculatory function in 9 patients with peripheral atherosclerotic arteriopathy (PAA) and in healthy control subjects were studied. ET-1 levels and microcirculatory function were evaluated both under basal conditions and 30, 60, and 90 min after acute administration of cloricromene (30 mg i.v.). PAA patients had significantly increased levels of ET-1 and impaired vascular parameters (studied by means of Winsor's Index, Gosling's Index, postischemic perfusion index and recovery time) when compared to control subjects. The acute administration of cloricromene (30 mg i.v.) did not change plasma ET-1 both in control subjects and in patients with PAA. In contrast, cloricromene produced a significant improvement in the postischemic perfusion index and in recovery time in arteriopathic patients. Control subjects and patients with PAA also underwent a cold pressor test (CPT) under basal conditions and (72 h later) 30 min after an acute intravenous administration of cloricromene (30 mg i.v.). CPT caused a higher increase in ET-1 in the patients with PAA compared to the control group, and a reduction in the vascular flow at the femoral level, while the pretreatment with cloricromene prevented both the increase in the levels of ET-1 and the reduction of the femoral vascular flow observed after the cold stimulus in patients with PAA. Our data show that cloricromene, besides ameliorating the microcirculatory function, is able to interfere with dynamic mechanisms, such as those induced by the CPT, capable of stimulating the release of ET-1 at the vascular level.

HLA-haploidentical umbilical cord blood stem cell transplantation in a child with advanced leukemia: clinical outcome and analysis of hematopoietic recovery.

Growing attention has been focused on cord blood as a source of transplantable hematopoietic stem cells. However, clinical experience is rather limited. In this study we describe a child with advanced acute lymphoblastic leukemia who received an HLA-haploidentical cord blood transplant. The patient was transplanted in third complete remission after conditioning with fractionated total body irradiation, thiotepa and cyclophosphamide. Forty-one milliliters of cryopreserved umbilical cord blood, containing 0.15 x 10(8) nucleated cells/kg and 0.25 x 10(4) CFU-GM/kg, were infused. Cyclosporine and prednisone were administered for graft-versus-host disease (GVHD) prophylaxis. The patient received G-CSF from day +1 to day +35, but no improvement in granulocyte counts was observed. Therefore, administration of GM-CSF was started on day +36 to day +59, which resulted in a significant increase in white blood cells and granulocyte counts. Sustained myeloid engraftment was evidenced by a granulocyte count > 0.5 x 10(9)/l by day +41. The presence of donor-derived cells could be documented in the peripheral blood and bone marrow of the patient by cytogenetic analysis, HLA phenotyping and DNA studies. Forty-one days after transplant, clonogenic bone marrow assays showed the presence of low frequencies of primitive hematopoietic progenitor cells (BFU-E = 19/10(5) and CFU-GM = 8/10(5)). The chimerism was complete and no host-derived cells could be detected. However, the engraftment was restricted to the myeloid lineage whereas lymphoid and megakaryocytic engraftments were inadequate. The immunophenotype of the patient's peripheral blood showed the presence of T lymphocytes expressing an immature phenotype (CD2+ CD3-) at day +21.(ABSTRACT TRUNCATED AT 250 WORDS)