ABSTRACT
This corrects the article DOI: 10.1038/leu.2015.246.
ABSTRACT
Children with acute lymphoblastic leukemia (ALL) and high minimal residual disease (MRD) levels after initial chemotherapy have a poor clinical outcome. In this prospective, single arm, Phase 2 trial, 111 Dutch and Australian children aged 1-18 years with newly diagnosed, t(9;22)-negative ALL, were identified among 1041 consecutively enrolled patients as high risk (HR) based on clinical features or high MRD. The HR cohort received the AIEOP-BFM (Associazione Italiana di Ematologia ed Oncologia Pediatrica (Italy)-Berlin-Frankfurt-Münster ALL Study Group) 2000 ALL Protocol I, then three novel HR chemotherapy blocks, followed by allogeneic transplant or chemotherapy. Of the 111 HR patients, 91 began HR treatment blocks, while 79 completed the protocol. There were 3 remission failures, 12 relapses, 7 toxic deaths in remission and 10 patients who changed protocol due to toxicity or clinician/parent preference. For the 111 HR patients, 5-year event-free survival (EFS) was 66.8% (±5.5) and overall survival (OS) was 75.6% (±4.3). The 30 patients treated as HR solely on the basis of high MRD levels had a 5-year EFS of 63% (±9.4%). All patients experienced grade 3 or 4 toxicities during HR block therapy. Although cure rates were improved compared with previous studies, high treatment toxicity suggested that novel agents are needed to achieve further improvement.
Subject(s)
Antineoplastic Combined Chemotherapy Protocols/administration & dosage , Hematopoietic Stem Cell Transplantation , Precursor Cell Lymphoblastic Leukemia-Lymphoma/drug therapy , Precursor Cell Lymphoblastic Leukemia-Lymphoma/therapy , Adolescent , Antineoplastic Combined Chemotherapy Protocols/adverse effects , Asparaginase/administration & dosage , Asparaginase/adverse effects , Child , Child, Preschool , Combined Modality Therapy , Cyclophosphamide/administration & dosage , Cyclophosphamide/adverse effects , Cytarabine/administration & dosage , Cytarabine/adverse effects , Daunorubicin/administration & dosage , Daunorubicin/adverse effects , Female , Humans , Infant , Kaplan-Meier Estimate , Male , Mercaptopurine/administration & dosage , Mercaptopurine/adverse effects , Methotrexate/administration & dosage , Methotrexate/adverse effects , Precursor Cell Lymphoblastic Leukemia-Lymphoma/mortality , Prednisone/administration & dosage , Prednisone/adverse effects , Prospective Studies , Remission Induction , Risk Factors , Transplantation, Homologous , Treatment Outcome , Vincristine/administration & dosage , Vincristine/adverse effectsABSTRACT
A 2-year-old girl presented to casualty with a right knee effusion after apparently minor trauma. Inflicted injury was suspected and full forensic coagulation studies were performed which revealed a mild deficiency of factor VIII. Screening of the exons and intron/exon boundaries of F8 gene indicated that the child appeared to be homozygous for the missense mutation c.5123G>A (p.Arg1708His) in exon 14 of the F8 gene. This mutation has been reported to be associated with mild haemophilia A. The possibility of hemizygosity had been masked by the test kit employed but referral to the genetics service and subsequent array CGH resulted in a diagnosis of Turner syndrome.
Subject(s)
Hemophilia A/diagnosis , Turner Syndrome/diagnosis , Child, Preschool , Chromosomes, Human, X , Diagnosis, Differential , Exons , Factor VIII/genetics , Female , Hemizygote , Hemophilia A/complications , Hemophilia A/genetics , Homozygote , Humans , Knee Injuries/diagnosis , Mutation , Turner Syndrome/complications , Turner Syndrome/geneticsABSTRACT
Classification of vasculitis remains unsatisfactory. This is largely because the pathogenetic mechanisms of this family of related disorders have not been fully understood. Existing classification criteria are useful but limited. This has become more apparent with the advent of more effective and more specific therapies. A rational basis for classification could significantly improve our approach to treatment. The development of diagnostic criteria in vasculitis is an even greater challenge but may ultimately provide more useful for the non-specialist clinician. International efforts are underway to provide more effective classification and diagnostic criteria.
Subject(s)
Vasculitis/classification , Humans , Vasculitis/diagnosis , Vasculitis/therapyABSTRACT
Factor replacement with BIOSTATE(®), a factor VIII (FVIII)/von Willebrand factor concentrate, forms the mainstay of treatment for children with von Willebrand disorder (VWD) in Australia and New Zealand. However, published data on the clinical efficacy and safety of BIOSTATE in the VWD paediatric population are limited. We retrospectively assessed the efficacy and safety of BIOSTATE in 43 children with VWD who received treatment for surgery, non-surgical bleeds or continuous prophylaxis at eight paediatric haemophilia centres in Australia and New Zealand. Data were collected on patient demographics, disease history, treatment history, dosage, administration, adverse reactions, concomitant medications and excessive bleeding events. BIOSTATE provided excellent/good haemostatic efficacy in 90% of surgical procedures (n = 42) with a mean daily FVIII dose of 47 IU FVIII:C kg(-1) and a median treatment duration of 3 days. Excellent/good haemostatic efficacy was achieved in 94% of non-surgical bleeding events (n = 72) with a mean FVIII dose of 45 IU FVIII:C kg(-1) day(-1) and a median treatment duration of 1 day. There were no bleeding events attributable to lack of efficacy. One case of nausea, possibly related to BIOSTATE administration, was reported. These results suggest that BIOSTATE is safe and effective for the treatment and prophylaxis of bleeding in children with VWD.
Subject(s)
Factor VIII/therapeutic use , Hemorrhage/prevention & control , Hemostatics/therapeutic use , von Willebrand Diseases/drug therapy , von Willebrand Factor/therapeutic use , Adolescent , Child , Child, Preschool , Drug Combinations , Female , Hemostasis, Surgical/methods , Humans , Infant , MaleABSTRACT
OBJECTIVE: To review and analyze the management of patients presenting with acute testicular torsion. DESIGN AND METHODOLOGY: A retrospective review of the medical records of all patients with a confirmed diagnosis of acute testicular torsion and all related presentations of acute scrotal pain was undertaken at the Princess Margaret Hospital from 1987 to 1997. RESULTS: The Accident and Emergency (A/E) Department logged 264 cases of acute scrotal pain (0.05 percent of all visits) of which 186 cases were diagnosed clinically and treated as acute epididymo-orchitis. Seventy-eight were admitted (55 percent were aged 11 to 20 years) and managed specifically as acute testicular torsion until proven otherwise by scrotal exploration. Fifty-five percent presented to the A/E services more than 12 hours after the onset of the pain. Seventy-seven cases were explored; 51 were confirmed as testicular torsion, of which 31 testes (61 percent) were not viable and underwent an orchiectomy. Within 6 hours of reaching the hospital, 58 percent of patients underwent exploration and 58 percent of these had a successful viable testicle and had orchiopexy. Of those explored after 6 hours (42 percent), only 22 percent had salvageable testes. An additional 13 patients were missed torsions. In total, there were 64 cases of confirmed testicular torsion, 69 percent had an orchiectomy; 31 percent had viable testes and were orchiopexed. CONCLUSION: Despite the maxim that acute testicular pain is torsion until proven otherwise, a significant number of cases suffer medical mismanagement. A risk management initiative needs to be directed to all physicians and a public awareness programme should be implemented.(Au)
Subject(s)
Child , Humans , Male , Adolescent , Spermatic Cord Torsion , Retrospective Studies , Spermatic Cord Torsion/diagnosisABSTRACT
The recent advent of laparoscopic splenectomy for the treatment of refractory idiopathic thrombocytopenic purpura (ITP) has been embraced by surgeons and hematologists in many institutions. However, the occurrence of accessory splenic tissue in a proportion of such splenectomies, either concurrently or later, raises the question of how to deal with this problem when it arises. We report that the laparoscopic approach, facilitated by lateral positioning, can be successfully used for the treatment of an accessory spleen causing recurrent ITP. The use of intraoperative nuclear imaging can greatly aid the localization and provide confirmation of complete excision of the nuclear focus, especially for a very small accessory spleen.
Subject(s)
Laparoscopy , Splenectomy/methods , Splenosis/diagnostic imaging , Adult , Female , Follow-Up Studies , Humans , Intraoperative Period , Purpura, Thrombocytopenic, Idiopathic/surgery , Radionuclide Imaging , Recurrence , Reoperation , Splenosis/surgeryABSTRACT
Itraconazole is a new orally active antifungal agent shown to have in vitro and experimental activity against Aspergillus spp. This case report documents the successful eradication of biopsy-proven invasive pulmonary aspergillosis in a 17 year old boy with acute lymphocytic leukaemia. Cerebral involvement by the fungal infection was suspected clinically but was not biopsy proven. Although the patient subsequently died following bone marrow transplant and Escherichia coli septicaemia there was no evidence of residual Aspergillus at autopsy.