A specifically pediatric-dedicated and -experienced team faces only limited difficulties when treating children with GVHD by ECP. Overall, ECP is efficient and well tolerated. Our experience was therefore pooled together with available pediatric data to establish clinical practice guidelines. These guidelines consider ECP as a first-line therapy in Grade IV aGVHD (in association with conventional pharmacologic approaches) and limited cGVHD and as a second-line therapy in steroid-resistant Grades II to III aGVHD and extensive cGVHD.
This simple and less expensive cryopreservation procedure can produce successful engraftment, comparable to that obtained with the standard storage procedure.
Articleshaematologica | 2013; 98 (7) 1089Corticosteroid can induce osteonecrosis in children with leukemia. Few studies have been designed to assess the influence of a wide range of cumulative steroid dose on this side effect. Prevalence, risk factors of symptomatic osteonecrosis and its impact on adults' Quality of Life were assessed in 943 patients enrolled in the French "Leucémies de l'Enfant et de l'Adolescent" (LEA) cohort of childhood leukemia survivors. During each medical visit, data on previous osteonecrosis diagnosis were retrospectively collected. Patients without a history but with suggestive symptoms were investigated with magnetic resonance imaging. The total steroid dose in equivalent of prednisone was calculated for each patient and its effect on osteonecrosis occurrence was studied in multivariate models. Cumulative incidence was 1.4% after chemotherapy alone versus 6.8% after transplantation (P<0.001). A higher cumulative steroid dose, age over ten years at diagnosis, and treatment with transplantation significantly increased the risk of osteonecrosis. A higher post-transplant steroid dose and age over ten years at time of transplantation were significant factors in the transplanted group. With patients grouped according to steroid dose quartile, cumulative incidence of osteonecrosis reached 3.8% in the chemotherapy group for a dose beyond 5835 mg/m 2 and 23.8% after transplantation for a post-transplant dose higher than 2055 mg/m 2 . Mean physical composite score of Quality of Life was 44.3 in patients with osteonecrosis versus 54.8% in patients without (P<0.001). We conclude that total and post-transplant cumulative steroid dose may predict the risk of osteonecrosis, a rare late effect with a strong negative impact on physical domains of Quality of Life.
Symptomatic osteonecrosis in childhood leukemia survivors: prevalence, risk factors and impact on quality of life in adulthood
The late effects and quality of life (QoL) in childhood acute leukemia survivors were compared between hematopoietic stem cell transplantation (HSCT) recipients and patients who underwent conventional therapy. The study included 943 patients, 256 of whom underwent HSCT (27.1%). Medical visits were conducted to detect the occurrence of physical late effects. Based on patient age, different questionnaires were used to assess QoL. To evaluate the association between HSCT and each type of late effect or QoL dimension, the appropriate multivariate regressions were performed. QoL mean scores were compared with those obtained for age- and sex-matched French control subjects. Of all the survivors, 674 (71.5%) had at least 1 late effect, with the risk being 5.0 CI95 (3.0-8.6) times higher for transplantation survivors. For child survivors, scoring of QoL showed no significant differences between the treatment groups. The adult HSCT survivors reported lower physical dimension QoL scores than chemotherapy survivors. Compared with French norms, the survivor group reported a significantly lower mental composite score; however, the physical composite score showed no significant difference. Thus, transplanted survivors have a high risk of developing late effects, resulting in a decreased physical well-being in adulthood. However, long after treatment completion, childhood leukemia survivors report that effects on psychological well-being are more important than they are in physical QoL dimensions.
The purpose of this multicenter study was to compare the long-term impact of a preparative regimen with either BUBU or TBI on health status and quality of life (QoL) in childhood acute leukemia survivors treated with hematopoietic SCT (HSCT). Two-hundred and forty patients were included. Sixty-six had received BU, while 174 had received TBI. Median follow-up from HSCT was 10.1 years. Multivariate analyses were performed to assess the occurrence of late effects according to treatment. QoL was assessed in 130 adults using SF-36 questionnaires. Patients developed fewer late complications after BU (2.35 vs 3.01, P ¼ 0.03) while the risk to present with at least one complication was equivalent in both groups (87.9% after BU and 93.1% after TBI, P ¼ 0.66). Detailed multivariate analyses revealed a lower risk of height growth failure (OR ¼ 0.2), cataract (OR ¼ 0.1) and iron overload (OR ¼ 0.2) after BU, and an increased risk of overweight (OR ¼ 3.9) and alopecia (OR ¼ 11.2). SF-36 mental and physical composite scores were similar in both treatment groups and proved significantly lower than French norms. Late effects induced by BU might differ from those experienced after TBI. Although less frequent, they are still of considerable importance and may affect patients' QoL.
The main aim of the Leucémies de l'Enfant et l'Adolescent (LEA) project (Childhood and Adolescent Leukaemia) is to study the determinants (medical, socioeconomic, behavioural and environmental) of medium- and long-term outcomes of patients treated for childhood acute leukaemia (AL). The LEA study began in 2004 and is based on a French multicentric prospective cohort. Included are children treated for AL since January 1980 (incident and prevalent cases), surviving at month 24 for myeloblastic AL and lymphoblastic AL grafted in first complete remission or at month 48 for lymphoblastic AL not grafted in first complete remission. Information is collected during specific medical visits and notably includes the following data: socioeconomic data, AL history, physical late effects (such as fertility, cardiac function and metabolic syndrome) and quality of life. Data are collected every 2 years until the patient is 20 years old and has had a 10-year follow-up duration from diagnosis or last relapse. Thereafter, assessments are planned every 4 years. In active centres in 2013, eligible patients number more than 3000. The cohort has already included 2385 survivors, with rate of exhaustiveness of almost 80%. Data access can be requested from principal coordinators and must be approved by the steering committee.
Summary:ings is unknown at present, some centers currently prefer to transplant these patients with PBSCs that have been depleted of potentially contaminating tumor cells. One To evaluate the feasibility and efficacy of CD34 ؉ cell immunoselection from routine peripheral blood stem possible method of reducing tumor cell contamination of the PBSC preparations is the positive selection of CD34 ϩ cell (PBSC) harvests in very small children a prospective study was performed in 15 children with advanced hematopoietic progenitor cells. 3 Initial trials of autografts with CD34 ϩ PBSCs have mainly been performed in adult neuroblastoma weighing 20 kg or less. Products of two consecutive leukaphereses carried out on a COBE Specpatients 4-6 and, outside of our team, 7 and so thus far there have been no reports of studies performed in small children. tra separator after G-CSF alone mobilization were pooled for immunoselection on Ceprate column. TheThe principal reasons for the restriction in the use of this technique in children with low body mass are PBSC collecmedian number of CD34 ؉ cells and total CFU-GM collected were respectively 5.9 ؋ 10 6 /kg (range 2.3-23.4) tion-associated difficulties (venous access, flow rate, metabolic and hemodynamic problems). In particular, when and 126.9 ؋ 10 4 /kg (range 52.9-559.9). After separation the median number of CD34 ؉ cells in the adsorbed fraclarge-scale collection is planned, which may require the processing of a considerable number of blood volumes, the tion was 2.6 ؋ 10 6 /kg (range 1-9.8) with a median purity of 54% (range 21-82) and a median of 95.7-fold safety of the procedure is of special importance. [8][9][10][11][12][13] We report the results of a prospective study designed to (range 35-250) enrichment. Thirteen patients underwent autografts with CD34 ؉ PBSCs after a busuldetermine whether sufficient numbers of CD34 ϩ cells can be obtained to provide reliable post-graft hematopoietic fan 600 mg/m 2 ؉ melphalan 180 mg/m 2 preparative regimen. The median number of days to achieve an reconstitution in very small children, after positive immunoselection of routinely collected leukaphereses products. absolute granulocyte count of 0.5 ؋ 10 9 /l and a platelet count of 20 ؋ 10 9 /l were respectively, 12 (range 10-24)We have investigated the feasibility and efficacy of this technique using the avidin-immunoaffinity column device and 35 (range 25-43). The median number of platelet transfusions was nine (range 2-15). We conclude that (Ceprate Stem Cell Concentrator; CellPro, Bothell, WA, USA) in 15 children with advanced neuroblastoma. These safe and effective immunoselection and transplantation of CD34 ؉ PBSC can be accomplished in children with patients may theoretically benefit from the isolation of CD34 ϩ hematopoietic progenitors, since the target CD34 low body mass.
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