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1.
Pediatr Hematol Oncol ; 33(7-8): 423-437, 2016.
Article in English | MEDLINE | ID: mdl-27960645

ABSTRACT

Hodgkin's lymphoma (HL) in children and adolescents is highly curable, but children are at risk of long-term toxicity. The MDH-03 guidelines were established in order to decrease the burden of treatment in good-responder patients, and this report should be considered a step toward further optimization of treatment within large collaborative trials. We report the therapy and long-term outcomes of 417 children and adolescents treated according to the national guidelines, which were applied between 2003 and 2007 in France. The patients were stratified into three groups according to disease extension. Chemotherapy consisted of four cycles of VBVP (vinblastine, bleomycin, VP16, prednisone) in localized stages (G1/95 pts/23%), four cycles of COPP/ABV (cyclophosphamide, vincristine, procarbazine, prednisone, adriamycin, bleomycin, vinblastine) cycles in intermediate stages (G2/184 pts/44%) and three cycles of OPPA (vincristine, procarbazine, prednisone, adriamycin) plus three cycles of COPP in advanced stages (G3/138 pts/33%). Radiation therapy of the involved field was given to 97% of the patients, with the dose limited to 20 Gy in good responders (88%). With a median follow-up of 6.6 years, the 5-year event-free survival (EFS) and overall survival (OS) were 86.7% (83.1-89.7%) and 97% (94.5-98.1%), respectively. EFS and OS for G1, G2, and G3 were 98% and 100%, 81% and 97%, and 87% and 95%, respectively. Low-risk patients treated without alkylating agents and anthracycline had excellent outcomes and a low expected incidence of late effects. Intensification with a third OPPA cycle in high-risk group patients, including stage IV patients, allowed for very good outcomes, without increased toxicity.


Subject(s)
Antineoplastic Combined Chemotherapy Protocols/administration & dosage , Hodgkin Disease/drug therapy , Hodgkin Disease/mortality , Adolescent , Child , Child, Preschool , Disease-Free Survival , Female , Follow-Up Studies , France , Hodgkin Disease/pathology , Humans , Male , Neoplasm Staging , Practice Guidelines as Topic , Survival Rate
3.
Pediatr Transplant ; 14(1): 109-14, 2010 Feb.
Article in English | MEDLINE | ID: mdl-19490483

ABSTRACT

We report results of RIC AHSCT in four adolescents with aggressive refractory HL. They all received three or four lines of therapy prior to RIC-AHSCT including autografts. At the time of RIC, they were in partial response except for one patient who had progressive chemoresistant disease. The conditioning regimen consisted of fludarabin, busulfan and ATG. They all had a matched related donor. The median follow-up was 12-16-month post-allograft. All patient transplants engrafted rapidly. The median time of hospitalization was 35 days. The median time to neutrophil recovery (>or=500/muL) was 19 days. All the patients were in complete donor chimerism at day 60. Four patients developed skin (grade

Subject(s)
Busulfan/therapeutic use , Hematopoietic Stem Cell Transplantation/methods , Hodgkin Disease/surgery , Myeloablative Agonists/therapeutic use , Vidarabine/analogs & derivatives , Adolescent , Antineoplastic Agents/therapeutic use , Drug Therapy, Combination , Female , Follow-Up Studies , Hodgkin Disease/diagnosis , Hodgkin Disease/drug therapy , Humans , Male , Positron-Emission Tomography , Severity of Illness Index , Time Factors , Transplantation, Homologous , Treatment Outcome , Vidarabine/therapeutic use
4.
Bone Marrow Transplant ; 40(9): 897-904, 2007 Nov.
Article in English | MEDLINE | ID: mdl-17704791

ABSTRACT

We compared late side effects and quality of life (QoL) in 430 survivors of childhood acute leukaemia based on whether they had undergone haematopoietic cell transplantation (n=142) or not (n=288). Mean age was 18.2 years and mean follow-up duration was 11.9 years. Multivariate logistic regression analyses were performed to compare the risk of each type of late effect in the two groups. Based on age, VSP-A or SF36 questionnaires were used to assess QoL. For each QoL dimension, multiple linear regression was done to construct models of association with the treatment group. Transplanted patients experienced more side effects, including height growth failure, gonadal dysfunction, hypothyroidism and cataract. Children and adolescents in the two treatment groups reported similar QoL levels for almost all dimensions except a better perception of school work by young transplanted children and more difficulties in relating to the medical staff for transplanted adolescents. In adults, two differences in physical domain of QoL were detected but the calculated effect sizes were less than 0.2 in each case, suggesting an uncertain clinical significance. In spite of a higher risk of physical adverse events in the transplanted group, very few clinically significant differences in QoL are detectable.


Subject(s)
Health Status , Hematopoietic Stem Cell Transplantation/adverse effects , Leukemia/complications , Leukemia/therapy , Survivors , Adolescent , Cataract/etiology , Child , Follow-Up Studies , Gonadal Disorders/etiology , Growth Disorders/etiology , Humans , Hypothyroidism/etiology , Linear Models , Surveys and Questionnaires
5.
Bone Marrow Transplant ; 39(8): 453-60, 2007 Apr.
Article in English | MEDLINE | ID: mdl-17334384

ABSTRACT

Cord blood (CB) units are increasingly used for allogeneic transplantation. Cell dose, a major factor for CB selection, is evaluated before freezing by each CB bank, using various techniques. This may introduce variability and affect the prediction of cell recovery after thawing, or haematopoietic reconstitution. Forty-two children were transplanted at the same institution with unrelated CB units. All units were thawed and evaluated at the same cell therapy facility, using standard procedures. We investigated: (i) factors that affect cell loss after thawing, and (ii) the importance of CD34(+) cell doses. Prefreeze and post-thaw CD34(+) cell doses were statistically correlated, thus suggesting that variability in numeration techniques used by different CB banks does not compromise the biological and clinical value of these figures. CD34(+) cell recovery appeared to be correlated with the absolute number of CD34(+) cells per frozen bag. Infused CD34(+) is the cell dose that better correlates with platelet reconstitution delay; in addition, when using a quartile comparison, haematopoietic recovery appeared to be related with prefreeze and post-thaw CD34(+) cell doses. We conclude that enumeration of CD34(+) cells in CB units is of biological significance, and may help select CB units and identify patients at risk of delayed recovery.


Subject(s)
Antigens, CD34/blood , Cord Blood Stem Cell Transplantation/methods , Hematopoietic Stem Cell Transplantation/methods , Neoplasms/therapy , Antigens, CD/blood , Cell Culture Techniques/standards , Child , Cord Blood Stem Cell Transplantation/standards , Fetal Blood , Hematopoietic Stem Cell Transplantation/standards , Humans , Immunosuppressive Agents/therapeutic use , Kinetics , Leukocyte Count , Platelet Count , Reproducibility of Results , Retrospective Studies , Transplantation Conditioning , Transplantation, Homologous , Whole-Body Irradiation
6.
Arch Pediatr ; 13(12): 1521-4, 2006 Dec.
Article in French | MEDLINE | ID: mdl-17010579

ABSTRACT

UNLABELLED: Thrombotic thrombocytopenic purpura (TTP), when accompanied by regenerative anaemia with schizocytosis, thrombopenia and neurological manifestations, is a disease whose main characteristic is the absence of the von Willebrand factor (vWF) cleaving protease. The two types of TTP are distinguishable by the presence or absence of antiprotease inhibitors, which are, respectively, either acquired or constitutional. The acquired autoimmune form is most frequently observed in adults. OBSERVATION: An adolescent with a previous history of moderate, isolated thrombopenia first showed symptoms of TTP at the age of 14. Positive antiprotease inhibitors in combination with a degeneration of protease activity confirmed the diagnosis of acquired autoimmune TTP. A treatment consisting of daily plasma exchange led to rapid improvement; however, a failed attempt to space out plasma exchanges necessitated the introduction of 4 weekly injections of Rituximab beginning on day 40, which was successful. Indeed, since the second injection of Rituximab on day 51, the number of platelets stabilized at a normal level, thereby allowing for the complete cessation of plasma exchange. At this writing - day 89 - the patient remains in persistent remission. CONCLUSION: Given the different therapeutic and prognostic implications of the 2 types of TTP in child patients, it is mandatory to end at an accurate biological diagnosis: whereas the constitutional form is effectively treated with plasma injections, the acquired form, while initially requiring plasma exchange, often necessitates the use of immunosuppressors during acute or relapse phase. The present study concerns a paediatric case of acquired TTP treated successfully with Rituximab during an acute dependant phase.


Subject(s)
Purpura, Thrombocytopenic, Idiopathic , Purpura, Thrombotic Thrombocytopenic , Remission Induction , Adolescent , Age Factors , Antibodies, Monoclonal/administration & dosage , Antibodies, Monoclonal/therapeutic use , Antibodies, Monoclonal, Murine-Derived , Humans , Immunologic Factors/administration & dosage , Immunologic Factors/therapeutic use , Male , Plasma Exchange , Purpura, Thrombocytopenic, Idiopathic/diagnosis , Purpura, Thrombocytopenic, Idiopathic/drug therapy , Purpura, Thrombocytopenic, Idiopathic/therapy , Purpura, Thrombotic Thrombocytopenic/diagnosis , Purpura, Thrombotic Thrombocytopenic/drug therapy , Purpura, Thrombotic Thrombocytopenic/therapy , Rituximab , Time Factors , Treatment Outcome
7.
Arch Pediatr ; 11(11): 1326-32, 2004 Nov.
Article in French | MEDLINE | ID: mdl-15519830

ABSTRACT

UNLABELLED: To evaluate the percentage and risk factors of thyroid dysfunction in 79 children who underwent bone marrow transplantation in a single centre. PATIENTS AND METHODS: The mean age of the cohort was 6.8 and mean follow-up 5.5 years. The 79 patients were divided in two groups according to the pretransplant conditioning regimen: fractionated total body irradiation (TBI)(N=54), chemotherapy with Busulphan (N=25). Thyroid function was evaluated by thyroid-stimulating hormone (TSH) and free thyroxine (fT4) tests. Overt hypothyroidism was defined by low fT4 blood levels and TSH > 4 mU/l, and compensated hypothyroidism by normal fT4 index and TSH >4 mU/L. RESULTS: The six-year probability of hypothyroidism was 36 +/-6% for the whole group of 79 patients, 49 +/-8% after TBI and 9 +/-6% in the Busulphan group (P <0.001). Neither gender, nor primary disease, nor presence of graft versus host disease were found to be statistically significant for occurrence of hypothyroidism in the TBI group. However, a younger age seemed to influence statistically the 6-year probability of hypothyroidism in the TBI group: 59 +/-9% if age <7.7 years versus 34 +/-13% if age >7.7 years (P =0.02). CONCLUSION: A careful follow-up of thyroid function is recommended even without TBI conditioning regimen. Young age as a potential risk factor of hypothyroidism has never been described and needs to be studied in a larger cohort.


Subject(s)
Bone Marrow Transplantation/adverse effects , Hypothyroidism/etiology , Adolescent , Age Factors , Child , Child, Preschool , Cohort Studies , Female , Humans , Infant , Male , Neoplasms/therapy , Risk Factors , Whole-Body Irradiation/adverse effects
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