Gemtuzumab ozogamicin (GO) monotherapy is reported to yield a 20-30% response rate in advanced acute myeloid leukaemia (AML). This study examined the efficacy and tolerability of GO combined with cytarabine (GOCYT) in children with refractory/relapsed CD33(+) AML. Seventeen children received GO 3 mg/m(2) on days 1, 4 and 7 plus cytarabine 100 mg/m(2)/d for 7 d on a compassionate-use basis. Seven patients then received GO-based consolidation. At the outset of GOCYT, two patients were refractory; eight patients were in refractory first relapse; six patients had relapsed after stem cell transplantation (SCT); and one patient [del(5q) therapy-related AML (t-AML)] had not yet been treated. Mean follow-up was 17 months (8-33 months). Ten responses were obtained after GOCYT induction, including complete remission (CR) or CR without complete recovery of platelets (CRp) in six patients (35%). The responses improved in three children who received GOCYT consolidation, increasing the CR + CRp rate to 53%. SCT was subsequently performed in eight responders. Grade 3-4 adverse events consisted of haematological disorders (n = 17, 100%) and documented infections (n = 5, 29%). No cases of sinusoidal obstructive syndrome occurred. Three patients were alive at the cut-off date for this analysis, all of whom had responded to GOCYT. GOCYT combination therapy yielded a high response rate (53%) and showed acceptable toxicity in heavily pretreated children with refractory/relapsed AML. These results warrant a larger prospective study.

International audienceOur purpose was to assess success rates in children of achieving optimal hematopoietic progenitor cells (HPCs) harvest after mobilization with 300 microg/kg pegfilgrastim. Between January 2005 and January 2007, 26 children with solid malignancies who were referred for HPC collection were consecutively included. Hematopoietic progenitor cell mobilization consisted of one s.c. injection of 300 microg/kg body weight (BW) of pegfilgrastim. The success criterion was defined as at least 5 x 10(6) CD34+ cells/kg during the first standard apheresis (less than 3 blood volumes processed (BVP)). After 26 inclusions, the Bayesian analysis gave a mean estimated success rate of 60.7% (95% credibility interval: 42.0-78.0%). The first apheresis allowed the collection of 8.3 x 10(6) CD34+ cells/kg BW (range 0.6-37.8), with a median of 2.8 BVP (range 1.4-3.0). Overall, the median of CD34+ cells collected was 12.4 x 10(6)/kg (range 2.7-37.8). The cumulative dose of anthracyclin was the only variable associated with the total number of CD34+ collected cells (P<0.05). Mobilization was clinically well tolerated in 20 patients. No drug-related adverse events of grade > or =3 occurred. We conclude that a single injection of 300 microg/kg pegfilgrastim in the hematological steady state is an efficient and well-tolerated method of HPC mobilization in children with solid malignancies

Traditional medicine (TM) status in population with access to modern medicine is not well clear in the whole country, even though the acceptance of TM is a well-established fact. TM has gained popularity in all regions of the developing world. This study is aimed at accessing the general knowledge, attitude, practice, the extent of use, benefits and safety of TM among people of Jos North L.G.A. A descriptive cross sectional study was carried out on 300 residents of Jos North L.G.A. A systematic random sampling was used to select households at intervals of six. Data was collected through the use of structured open and closed ended questionnaires and interview and the data were analysed using SPSS 20. 274 out of 300 questionnaires were valid. Among the respondents, 56.9% are between 19-28 years. 100% of the participants have good knowledge of TM, 62.8% had no adverse effect from the use of TM, while 70.1% do not agree that TM is safer than modern medicine. 70.4% of the participants plan to use TM in the future. 29.9% of the respondent prefer TM, 31.8% prefer modern medicine while 38.3% prefer both. The population in Jos North has good knowledge with high acceptability and use of TM. The main reasons for the high acceptability are due to lesser cost, availability, effectiveness and efficacy.

The use of Gemtuzumab-Ozogamicin (GO), an anti-CD33 monoclonal antibody, is associated to a 20–30% response rate in refractory/relapsed AML. We have reviewed the efficacy and the tolerance of a combination of GO plus cytarabine (GOCYT) given to children with refractory/relapsed AML in order to improve the response rate. This retrospective analysis includes 17 children (9M, 8F) who received the same induction schedule on a compassionate-use basis between may 2004 and june 2006: GO 3 mg/m2/d D1, D4, D7 plus cytarabine 100 mg/m2/d CIV for 7 days. In addition, 6 patients received a consolidation course with GO 3 mg/m2 D1 plus cytarabine 100 mg/m2/d D1 to D7 and 1 patient received GO 3 mg/m2 monthly for 4 doses. Median age at diagnosis of AML is 4.5 y (0.4–16.7). The patient distribution is: de novo AML: 13; biclonal (T and myeloid) acute leukemia: 1; erythremia: 1; t-AML: 2. Initial caryotype was unfavourable in 6 cases, intermediate in 9 and favourable in 2. Patient’s status at GOCYT start were as follows: 2 pts refractory to 1st line therapy (t-AML: 1, M7: 1); 8 pts in refractory first relapse (median time to relapse: 7.25 m, 5–19); 6 pts in relapse after BMT (median time to relapse: 13.5 m, 10.5–111); 1 untreated pt (del(5q) t-AML). CD33 expression was positive in all cases. The median time between initial diagnosis and GOCYT administration is 11.5 m (3–116). MFU is 5 months (1–21). Ten responses were obtained after GOCYT induction (59%): CR: 2, CRp: 4 and PR: 4. Noteworthy, 3 of 7 responders who received a consolidation course with GO improved their response (PR to CR: 2, PR to CRp: 1). Lastly, a subsequent BMT could be performed in 7 responders. Six pts were in leukemic failure and 1 pt died from invasive aspergillosis without bone marrow evaluation. Grade 3 and 4 side effects were: hematologic (17, 100%); documented infections (5): invasive aspergillosis (2 including one responder), pulmonary reactivation of aspergillosis (1), streptococcus oralis septicemia (1), candida kefyr septicemia (1); fever and neutropenia without documented infection (6); hyperbilirubinemia (1); hypertransaminasemia (1); sinusoidal obstruction syndrome SOS (0). Among the 7 patients alive at point date, 6 were responders to GOCYT. Conclusion: a high response rate (59%) was objectived in children with heavily pre-treated refractory/relapsed AML allowing a subsequent BMT in 7. A consolidation course improved the quality of the response in some cases. Despite the limitations of this study, the response rate seems higher than the one observed in phase I–II or compassionate-use pediatric studies using GO alone. No unexpected toxicity was seen. Remarkably, no SOS was observed despite previous BMT in 8 out of the 17 children and a second BMT was feasible in responding pts. GOCYT should be tested prospectively in a largest pediatric AML population.