Twelve-month prevalence of haemarthrosis and joint disease using the Haemophilia Joint Health score: evaluation of the UK National Haemophilia Database and Haemtrack patient reported data: an observational study.

Objectives: To report the 12-month prevalence of joint bleeds from the National Haemophilia Database (NHD) and Haemtrack, a patient-reported online treatment diary and concurrent joint disease status using the haemophilia joint health score (HJHS) at individual joint level, in children and adults with severe haemophilia A and B (HA/HB) without a current inhibitor.

Design: A 2018 retrospective database study of NHD from which 2238 cases were identified, 463 patients had fully itemised HJHS of whom 273 were compliant in recording treatment using Haemtrack.

Setting: England, Wales and Scotland, UK.

Treatment regimens and outcomes in severe and moderate haemophilia A in the UK: The THUNDER study.

Introduction: The THUNDER study provides an analysis of treatment patterns and outcomes in UK patients with severe or moderate haemophilia A (SHA/MHA) in 2015.

Methods: Patients with SHA or MHA registered with the UK National Haemophilia Database (NHD) were segregated by severity, inhibitor status and age. Haemophilia joint health score (HJHS) was derived from NHD records and treatment regimen and annualized bleed/joint-bleed rate (ABR/AJBR) from Haemtrack (HT) in HT-compliant patients.

Effect of gemtuzumab ozogamicin on acute myeloid leukemia blast cells in vitro, as a single agent and combined with other cytotoxic cells.

The effect of gemtuzumab ozogamicin (GO) alone, or combined with low-dose cytarabine or etoposide, on the proliferation of acute myeloid leukaemia blast cells in vitro was investigated. GO alone induced a dose-dependent inhibition of proliferation although an increase in apoptosis was only seen in a minority of patients. A correlation was found between PgP function and GO sensitivity but not between CD33 or PgP expression and GO.

IgA myeloma of donor origin arising 7 years after allogeneic renal transplant.

We report the case of a 38-year-old man who developed IgA myeloma of donor origin 7 years after allogeneic renal transplant. The diagnosis of multiple myeloma was unequivocal and based on positive results from serum electrophoresis, skeletal survey and bone marrow investigations. The donor origin of the myeloma cells was confirmed by DNA fingerprinting.

Effect on cell kill of addition of multidrug resistance modifiers cyclosporin A and PSC 833 to cytotoxic agents in chronic lymphocytic leukaemia.

Expression of P-glycoprotein (Pgp), the drug efflux pump which mediates multidrug resistance (MDR), has been widely reported in chronic lymphocytic leukaemia (CLL) and improved accumulation of daunorubicin has been reported using the MDR reversing agent cyclosporin A (CSA). We have investigated the effects on cell kill of the addition of CSA and its analogue PSC 833 to daunorubicin, doxorubicin, idarubicin, mitozantrone and fludarabine in samples from 51 patients with CLL using an MTT [3(4,5-dimethylthaizol-2-yl)-2,5-diphenyltetrazolium bromide] assay. Pgp expression was assessed by immunocytochemistry using the JSB-1 monoclonal antibody.

Overexpression of lung-resistance protein and increased P-glycoprotein function in acute myeloid leukaemia cells predict a poor response to chemotherapy and reduced patient survival.

We investigated the role of the drug resistance-related proteins LRP, MRP and Pgp and the apoptotic suppressor, bcl-2, in relation to other clinical characteristics, with respect to response and survival in 91 patients with newly diagnosed AML, treated with standard chemotherapy. Multivariate analysis showed that poor response to chemotherapy was associated with increasing age (P=0.0004), LRP expression (P=0.

Ethnicity study and non-selective screening for haemoglobinopathies in the antenatal population of central Manchester.

The objective of this study was to determine the frequency of ethnic groups within the antenatal population in central Manchester and thereby ensure that the haemoglobinopathy service was targeting the correct population and their needs. Ethnic data collection records of 6718 patients were analysed over a 7 month period. Of these 62.

Effect on cell kill of addition of multidrug resistance modifiers cyclosporin A and PSC 833 to cytotoxic agents in acute myeloid leukaemia.

Multidrug resistance (MDR) mediated by the drug efflux pump P-glycoprotein (Pgp), may cause remission failure and relapse in patients with acute myeloid leukaemia (AML) by extruding cytotoxic agents such as anthracyclines from leukaemic cells thus allowing them to survive. Cell line data suggest that reversal of MDR is possible using modifying drugs such as cyclosporin A (CSA) and its analogue PSC 833. We have investigated the effects on cell kill of the addition of CSA and PSC 833 to daunorubicin, idarubicin, mitozantrone, etoposide and cytarabine in 52 fresh cell samples from AML patients using an MTT assay.

Successful treatment of HTLV-1-associated acute adult T-cell leukaemia lymphoma by allogeneic bone marrow transplantation.

HTLV-1-associated acute adult T-cell leukaemia-lymphoma (ATL) is a highly aggressive malignant disorder with a median survival of 6 months or less. We describe an Afro-Caribbean female with very poor prognosis ATL who underwent chemotherapy with a 4 d infusion schedule of cyclophosphamide, doxorubicin and etoposide, followed by successful allogeneic bone marrow transplantation (BMT) from her HTLV-1-negative histocompatible sister. The patient remains in complete remission 23 months after BMT and has 100% donor haemopoiesis with no evidence of HTLV-1 infection on PCR testing.

Acute tumour lysis syndrome.

Acute tumour lysis syndrome results from a rapid massive release of cellular breakdown products consequent upon tumour cell death following effective therapy. This may overwhelm normal excretory mechanisms, resulting in metabolic disturbance which can lead to sudden death or prolonged morbidity from renal impairment.

High incidence of type II autoantibodies in pernicious anaemia.

Aims: To investigate the incidence of type II autoantibodies to intrinsic factor in pernicious anaemia.

Methods: Three hundred and forty four serum samples submitted for intrinsic factor antibody (IFAB) analysis on clinical or laboratory grounds were tested by an established radioassay and a new enzyme linked immunosorbent assay (ELISA) method for type I and total IFAB, respectively. Sixty of these were found to be positive by ELISA; this method was used to test further, 40 samples of adequate volume for types I and II antibodies.

Effects of azathioprine on response of renal anaemia to subcutaneous recombinant human erythropoietin.

Aims: To determine the effect of concomitant azathioprine treatment on the response of patients with renal failure to treatment with subcutaneous recombinant human erythropoietin (r-HuEPO).

Methods: Two groups of patients with renal failure not receiving haemodialysis were studied. One comprised seven patients receiving erythropoietin alone, the second consisted of nine patients who were also treated with azathioprine.

Comparative ferrokinetic study with initial and extended iron clearance models.

Erythrokinetic studies were performed on 10 patients with chronic myelofibrosis and 11 patients with myelodysplasia (MDS). Values for plasma iron turnover, marrow iron turnover, and erythron transferrin uptake were derived using two ferrokinetic models. One entailed analysis of the extended plasma iron clearance over a number of days, the other comprised analysis of the initial plasma iron clearance during the first few hours of the study.

Detection of erythroid hypoplasia in myelofibrosis using erythrokinetic studies.

The iron kinetic model described by Ricketts et al was used to study haemopoiesis in chronic myelofibrosis. The clearance of 59Fe-labelled transferrin from the plasma was analysed to quantify total, effective, and ineffective erythropoiesis, denoted by the terms marrow iron turnover (MIT), red cell iron turnover (RCIT), and per cent ineffective iron turnover (IIT%), respectively, in 12 cases of this disease. The patterns obtained were variable: values for MIT ranged from 24.