In the absence of head-to-head clinical data, the objective of this study was to indirectly compare the efficacy and safety of a bivalirudin-based anticoagulation strategy with that of heparin monotherapy in patients with ST-elevation myocardial infarction (STEMI) intended for primary percutaneous coronary intervention. A systematic literature review was performed to identify randomized controlled trials to build a network of bivalirudin and heparin monotherapy strategies in STEMI patients using heparin, with glycoprotein IIb/IIIa inhibitor as a common reference strategy. At 30 days, the bivalirudin-based strategy was expected to result in lower mortality rates than heparin monotherapy (odds ratio [OR], 0.55; credible limit [CrL], 0.32–0.95). This relationship was sustained at 1 year. At 30 days, the risk for stroke (OR, 0.88; CrL, 0.37–2.13), myocardial infarction (OR, 0.79; CrL, 0.40–1.55), and thrombolysis in myocardial infarction major and minor bleedings (OR, 0.66; CrL, 0.45–0.98) tended to be numerically reduced with bivalirudin in comparison with heparin monotherapy. For patients with STEMI intended for primary percutaneous coronary intervention, bivalirudin is associated with lower mortality rates in comparison with heparin monotherapy. This study suggests that bivalirudin is more effective and safer than heparin monotherapy and should therefore be preferred over heparin monotherapy.
primary angioplasty; STEMI; pharmacology
Vitamin D deficiency and anemia are common in the Middle East, and vitamin D deficiency and hyperparathyroidism have been reported to be associated with an increased prevalence of anemia. In this study, the hypothesis that vitamin D deficiency and hyperparathyroidism may be associated with anemia in a Bahraini population was tested. Association of hyperparathyroidism and vitamin D levels (deficiency and insufficiency) with anemia was investigated in 421 Bahrainis (213 males and 208 females). In females, the prevalence of anemia was significantly associated with vitamin D deficiency independent of parathyroid hormone levels (odds ratio: 2.9; 95% confidence interval: 2.3–10.5; P = 0.001). In females, the prevalence of anemia appeared to be significantly associated with hyperparathyroidism (odds ratio: 2.1; 95% confidence interval: 1.2–3.7; P = 0.01); however, this significant association disappeared when adjusted for vitamin D deficiency (odds ratio: 1.6; 95% confidence interval: 0.75–6.5; P = 0.154). Results from this study suggest that vitamin D deficiency is independently associated with anemia in females but not males. Further studies to determine whether vitamin D supplementation could be used to treat anemia are warranted.
vitamin D; deficiency; hyperparathyroidism; anemia
Recent advances in our understanding of the molecular mechanisms underlying hypereosinophilia have led to the development of a ‘molecular’ classification of myeloproliferative disorders with eosinophilia. The revised 2008 World Health Organization classification of myeloid neoplasms included a new category called “myeloid and lymphoid neoplasms with eosinophilia and abnormalities of PDGFRA, PDGFRB or FGFR1.” Despite the molecular heterogeneity of PDGFR (platelet-derived growth factor receptor) rearrangements, tyrosine kinase inhibitors at low dose induce rapid and complete hematological remission in the majority of these patients. Other kinase inhibitors are promising. Further discoveries of new molecular alterations will direct the development of new specific inhibitors. In this review, an update of the classifications of myeloproliferative disorders associated with hypereosinophilia is discussed together with open and controversial questions. Molecular mechanisms and promising results of tyrosine kinase inhibitor treatments are reviewed.
hypereosinophilia; classification; myeloproliferative disorders; molecular alterations; tyrosine kinase inhibitor
Deferasirox is a once-daily, oral iron chelator that is widely used in the management of patients with transfusional hemosiderosis. Several Phase II trials along with their respective extension studies as well as a Phase III trial have established the efficacy and safety of this novel agent in transfusion-dependent patients with β-thalassemia, sickle-cell disease and bone marrow-failure syndromes, including myelodysplastic syndrome and aplastic anemia. Data from various clinical trials show that a deferasirox dose of 20 mg/kg/day stabilizes serum ferritin levels and liver iron concentration, while a dose of 30–40 mg/kg/day reduces these parameters and achieves negative iron balance in red cell transfusion-dependent patients with iron overload. Across various pivotal clinical trials, deferasirox was well tolerated, with the most common adverse events being gastrointestinal disturbances, skin rash, nonprogressive increases in serum creatinine, and elevations in liver enzyme levels. Longer-term extension studies have also confirmed the efficacy and safety of deferasirox. However, it is essential that patients on deferasirox therapy are monitored regularly to ensure timely management for any adverse events that may occur with long-term therapy.
deferasirox; iron overload; thalassemia; sickle-cell disease; myelodysplastic syndrome
For many decades, the vitamin K antagonist warfarin has been the mainstay of treatment for
various conditions that require anticoagulation, including atrial fibrillation. Although the
efficacy of warfarin in both prevention and treatment of thrombosis has been demonstrated in
numerous randomized clinical studies, one of the major concerns that remains is the risk of
bleeding. Although the net benefit of warfarin has been demonstrated in large clinical trials,
physicians and patients alike are often reluctant to use warfarin because of the bleeding risk.
Bleeding in patients on warfarin is generally minor requiring no intervention, but the development
of a major bleeding complication is associated with significant morbidity and can even be fatal.
Numerous risk factors that increase the probability of having a hemorrhage while on warfarin have
been identified, and bleeding risk scores have been developed. Various strategies to reduce bleeding
risks have been developed and have become more important, since the use of warfarin and other
anticoagulants continues to increase. This paper provides a concise review of bleeding risk factors,
while outlining recommendations both physician and patients can incorporate to help reduce the risk
hemorrhage; warfarin; thrombosis; anticoagulants; dabigatran; vitamin K antagonist
Peripheral neuropathy (PN) caused by bortezomib is an important complication of multiple myeloma. Subcutaneous injection of bortezomib reduced PN, but 24% of cases were grade 2 PN and 6% of cases were grade 3 PN. PN higher than grade 2 was not resolved by subcutaneous injection. PN higher than grade 3 has serious dose limiting toxicity and is the cause of discontinuing bortezomib treatment. Lafutidine is an H2-blocker with gastroprotective activity and is thought to function by increasing mucosal blood flow via capsaicin sensitive neurons. The same activity of lafutidine is considered to improve glossodynia and taxane induced PN. We hypothesized that lafutidine prevents or improves PN that is caused by bortezomib. In the current study, bortezomib was administered in the usual manner (intravenous administration of bortezomib 1.3 mg/m2, twice a week for 2 weeks, followed by 1 week without treatment) for up to four cycles to compare our data with other studies. Lafutidine was administered orally at a dose of 10 mg twice daily. In our eight evaluated cases, the total occurrence of PN was four out of eight patients (50%). There were only grade 1 PN (4 out of 8) cases, and no cases higher than grade 2. We conclude that (1) the total occurrence of PN was not improved, (2) there was no PN after the first course, (3) there were only grade 1 cases and there were no cases higher than grade 2, and (4) no cases discontinued bortezomib treatment because of PN. This is the first report showing that lafutidine is useful for the amelioration of bortezomib induced PN.
bortezomib induced peripheral neuropathy; lafutidine; capsaicin sensitive neurons; calcitonin gene-related peptide; transient receptor potential 1; multiple myeloma
Apart from challenging the bone marrow to increase its red cell production, thereby producing more blood for the donor, regular blood donation has been shown to have several benefits, one of which is preventing accumulation of body iron which can cause free radical formation in the body. This study was carried out to assess body iron stores in regular blood donors.
A total of 52 regular (study) and 30 first-time (control) volunteer blood donors were studied prospectively. Twenty milliliters of venous blood was drawn from each subject, 5 mL of which was put into sodium ethylenediamine tetra-acetic acid specimen bottles for a full blood count, including red blood cell indices. The remaining sample was allowed to clot in a plain container, and the serum was then retrieved for serum ferritin, serum iron, and serum transferrin receptor measurement by enzyme-linked immunosorbent assay.
Mean hemoglobin and packed cell volume in the study group (13.47 ± 2.36 g/dL and 42.00 ± 7.10, respectively, P = 0.303) were not significantly higher than in the control group (12.98 ± 1.30 g/dL and 39.76 ± 4.41, respectively, P = 0.119). Mean serum ferritin was 102.46 ± 80.26 ng/mL in the control group and 41.46 ± 40.33 ng/mL in the study group (P = 0.001). Mean serum ferritin for women in the study group (28.02 ± 25.00 ng/mL) was significantly lower than for women in the control group (56.35 ± 34.03 ng/mL, P = 0.014). Similarly, men in the study group had a lower mean serum ferritin (48.57 ± 45.17 ng/mL) than men in the control group (145.49 ± 87.74 ng/mL, P = 0.00). The mean serum transferrin receptor value was higher in the study group (1.56 ± 0.88 μg/mL) than in the control group (1.19 ± 0.38 μg/mL, P = 0.033).
These findings suggest that hemoglobin concentration, packed cell volume, and serum iron levels are not significantly affected by regular blood donation and that regular blood donors appear to have reduced iron stores compared with controls.
blood donors; hemoglobin; serum ferritin; serum iron; transferrin receptors
Congenital Factor XIII (FXIII) deficiency is a rare, inherited, autosomal recessive coagulation disorder. Most mutations of this condition are found in the A-subunit with almost half these being missense mutations. Globally, approximately one in three million people suffer from this deficiency. Factor XIII deficiency is associated with severe life threatening bleeding, intracranial hemorrhage, impaired wound healing, and recurrent pregnancy losses. FXIII is known to have a potential role in mediating inflammatory processes, insulin resistance, bone metabolism, neoplasia, and angiogenesis. The algorithm provided for FXIII diagnosis and classification will enable prompt identification and early intervention for controlling potential life threatening complications. Prophylactic replacement therapy using blood products containing FXIII such as fresh frozen plasma, cryoprecipitate, or using FXIII concentrate remains the mainstay for the management of FXIII deficiency. In most parts of the world, cryoprecipitate and plasma transfusions are the only treatments available. Management developments have revealed the effectiveness and safety of recombinant FXIII concentrate for prophylaxis and treatment. The aim of this review is to provide an overview of advancements made in the management of FXIII deficiency from the time it was first detected, highlighting novel developments made in recent years. Greater research is warranted in identifying novel approaches to manage FXIII deficiency in light of its underlying pathophysiology.
Factor XIII; Factor XIII deficiency; treatment; diagnosis; inherited coagulative disorders
Serum ferritin is considered to be one of the most important tools in the measurement of iron balance in steady-state sickle cell disease. Increased gastrointestinal absorption of iron has been reported in sickle cell disease because of the associated chronic hemolysis, and it is also thought that repeated red cell transfusion consequent to chronic hemolysis and anemia causes excessive iron levels. The aim of this study was to determine overall and gender-specific mean ferritin levels in patients with steady-state sickle cell disease in order to establish the prevalence of iron deficiency and overload.
This was a cross-sectional study in homozygous patients with sickle cell disease attending the sickle cell clinic at Lagos State University Teaching Hospital, Ikeja. A 5 mL blood sample was collected in plain bottles from consenting participants during steady-state periods. The serum was separated and analyzed for ferritin by enzyme-linked immunosorbent assay. Another 5 mL sample was collected for a full blood count, done on the same day of collection, to determine red blood cell indices, ie, mean cell volume, mean cell hemoglobin concentration, and mean corpuscular hemoglobin concentration. The Pearson Chi-square test was used for statistical analysis. The differences were considered to be statistically significant when P was <0.05.
In total, 103 patients were recruited for this study and comprised 58 women (56.40%) and 45 men (43.70%). The overall mean ferritin concentration was 93.72 ± 92.24 ng/mL. The mean ferritin concentration in the women was 92.00 ± 88.07 ng/mL and in men was 96.41 ± 99.80 ng/mL. Only eight (7.76%) of the 103 patients had a serum ferritin level < 15 ng/mL, while two subjects (1.94%) had a serum a ferritin level > 300 ng/mL. Ninety-three subjects (90.29%) had serum ferritin within the normal reference range of 15–300 ng/mL.
In this study, 90% of subjects with sickle cell disease had normal iron stores; serum ferritin was higher in men than in women, and iron deficiency was more common than overload in the disease.
serum ferritin levels; sickle cell disease
Early treatment of bleeds in hemophilia patients, both with and without inhibitors, has been shown to be of immense benefit in the overall clinical outcome. Despite the advantages of treating the bleeding episodes early, significant barriers and limitations remain. The aim of this review is to highlight the various barriers and perceived limitations to early therapy of bleeding episodes, especially in patients who have developed inhibitors to factor VIII. The peer-reviewed literature was searched for articles on hemophilia patients, with and without inhibitors, and early treatment, to identify the barriers to early treatment and potential impact on patient outcomes. The most important barrier is the educational barrier, which involves lack of awareness among patients regarding the signs of a bleed, as well as importance of early therapy. It is also common for parents or caregivers of school-age children to exhibit inconvenience and scheduling barriers. Distance to the treatment center can also play a role here. Some patients experience financial barriers related to cost of clotting factor products, insurance coverage, or insurance caps and out-of-pocket costs. Rarely, there can also be problems related to venous access or home infusion. Lastly, multiple psychosocial barriers can prevent adherence to treatment regimens. Identification and addressing these individual barriers will result in improved compliance rates, prevent joint damage, be more cost-effective, and lead to better overall health of these patients.
hemophilia A; hemophilia B; inhibitors; outcomes; quality of life; cost of care
A 77-year-old male was admitted to hospital after suffering a pelvic bone fracture in a road traffic accident and was incidentally found to have IgG-kappa-type multiple myeloma with hypercalcemia. The patient was also noted to be hypokalemic and had low HCO3−, with possible damage to the distal tubules in the kidneys. When the treatment was begun with bortezomib/dexamethasone/elcatonin and sodium bicarbonate (NaHCO3) in normal saline (equivalent to a daily sodium dose of 200 millimoles per liter [mmol/L]), the patient was in a state of poor oral fluid intake. The patient developed hypernatremia and hyperchloremia, with a peak serum sodium and chloride levels of 183 mmol/L and 153 mmol/L, respectively, at the sixth day after the start of treatment. Following the switch of the intravenous infusions from normal saline to soldem 1 and soldem 3 solutions, these high-electrolyte levels gradually returned to normal over the next 7 days. Although the patient showed disturbed consciousness (Japan Coma Scale = JCS-I-3) during the period of electrolyte abnormality, he eventually fully recovered without sequelae. In this patient, we successfully managed the severe hypernatremia/hyperchloremia, caused by the combined effects of intravenous saline burden in a state of poor oral fluid intake, during the treatment for IgG-kappa type multiple myeloma.
hypernatremia; hyperchloremia; multiple myeloma; IgG-kappa; metabolic acidosis; renal tubular damage
A few reports have linked regular blood donation to the lowering of parameters of lipid profile. Estimating the lipid profile is an accepted method of assessing an individual’s risk for coronary heart disease, particularly if there is evidence of lipid peroxidation. Regular blood donation may lower iron stores, and this in turn lowers lipid peroxidation. This study was carried out to determine the effect of blood donation on lipid profile.
Materials and methods
Eighty-two participants consented to participate and were enrolled into the study, 52 of whom were regular blood donors (study group) and 30 were non-donors (control group). Venous blood (10 mL) was drawn from each subject into new plain screw-capped disposable plastic tubes. This was allowed to clot and the serum was used to determine total cholesterol, triglycerides, low-density lipoprotein, and high-density lipoprotein.
The mean total cholesterol (4.66 ± 0.86 mmol/L), triglycerides (1.22 ± 0.64 mmol/L), and low-density lipoprotein (2.32 ± 0.73 mmol/L) were significantly lower in the regular blood donors than the control group (5.61 ± 1.26 mmol/L, 1.77 ± 2.9 mmol/L, and 3.06 ± 0.89 mmol/L, respectively; P < 0.05 in all cases). Also, while 42% of the study group had a low/high-density lipoprotein ratio of at least three, about 57% of the control group had a ratio of at least three (P = 0.21).
Regular blood donation may be protective against cardiovascular disease as reflected by significantly lower mean total cholesterol and low-density lipoprotein levels in regular blood donors than in non-donors.
donor; total cholesterol; low-density lipoprotein
In northeastern Italy, according to Italian legislation, authorized public facilities can accept the donation and preservation of cord blood stem cells (CB-SC). Attitudes and knowledge in pregnant women differs between the local and immigrant (non-European Union [EU]) population. In this study we assessed the choices that pregnant women have with respect to the public and private harvesting system and the main reasons driving their decisions. We examined the ethnic origin of the families and compared tests for syphilis screening and leukocyte (WBC) counts in the CB-SC bags that are required for validation of the collection.
Out of a population of 3450 pregnant patients at the Institute for Maternal and Child Health of Trieste, northeast Italy, 772 women agreed to cord blood harvesting and the associated lab tests. Of these, 221 women (28.6%) were from immigrant families of non-EU countries. Their ethnic affiliation was recorded, and tests were performed for syphilis screening and for nucleated red blood cell (NRBC) interference with the WBC count in CB-SC bags to assess cellularity and to determine if storage was appropriate.
Of the 772 pregnant women, 648 (84.0%) accessed the public collection system, which is free of charge, and 124 (15.0%) accessed the private fee-based system. One woman from the non-EU group opted for the private fee-based system. Of the 3450 pregnant women screened for syphilis at the Institute for Maternal and Child Health, the Treponema pallidum hemagglutination (TPHA) and Venereal Disease Research Laboratory (VDRL) tests were the main tests performed (66.0% of total cases) because many gynecologists in the public harvesting system apply the Italian regulations of the 1988 Decree, while the private system requires tests on syphilis and leaves the option to the lab physicians to select the best determination method. We found that the chemiluminescence method was more specific (97.0%) than the TPHA (83.0%) and nontreponemal rapid plasma reagin VDRL (75.0%) tests (P < 0.05, χ2 test). The specificity link between the two automatic methods versus microscopes for WBC dosing and NRBC interference was r2 = 0.08 (ADVIA 120) and r2 = 0.94 (XE-2100). The public system does not include human T-cell lymphotropic virus testing; this is reserved for the population from endemic zones.
In northeastern Italy current legislation prevents the establishment of private fee-based banks for storage of CB-SC. The cryopreservation, for future autologous personal or family use, is possible only by sending to foreign private banks, with a further fee of €300. These regulations confirm that Italian legislation tries to increase the anonymous allogenic donations and the number of CB-CS bags stored in the free-cost public system, that are available to anyone with therapeutic needs. Private banking is used almost exclusively by the wealthier local population. In the public system, many physicians continue to use older Italian laws regarding syphilis diagnosis, and NRBC interference on WBC count may have an impact on cord blood harvesting. Our findings suggest that in the EU there is no consensus policy on donor management. The value of storage for potential use within the family is useful only with collaboration between the public and the private systems.
cord blood collection; public system; private system; pregnant women’s choice
The World Health Organization defines anemia as the point at which the amount of hemoglobin in the circulation falls below World Health Organization cutoffs for specific age and sex groups. Anemia is a worldwide problem of complex etiology and is associated with many factors. The purpose of this review was to describe the biomarkers used to identify the nature of anemia in patients and in the community. The important biomarkers are the automated red cell counts, tests for nutritional deficiencies, hemoglobinopathies, and inflammation. Diseases are important potential initiators of anemia, but biomarkers of specific diseases are not included in this review, only the underlying feature common to all disease – namely, inflammation.
iron deficiency; biological markers; blood cell count; inflammation; avitaminosis; hemoglobinopathies
Toxic death is a big problem in the treatment of childhood acute lymphoblastic leukemia (ALL), especially in low-income countries. Studies of ciprofloxacin as single agent prophylaxis vary widely in success rate. We conducted a double-blind, randomized study to test the effects of ciprofloxacin monotherapy as prophylaxis for sepsis and death in induction treatment of the Indonesian childhood ALL protocol.
Patients were randomized to the ciprofloxacin arm (n = 58) and to the placebo arm (n = 52). Oral ciprofloxacin monotherapy or oral placebo was administered twice a day. All events during induction were recorded: toxic death, abandonment, resistant disease, and complete remission rate.
Of 110 patients enrolled in this study, 79 (71.8%) achieved CR. In comparison to the placebo arm, the ciprofloxacin arm had lower nadir of absolute neutrophil count during induction with median of 62 (range: 5–884) versus 270 (range: 14–25,480) × 109 cells/L (P < 0.01), greater risks for experiencing fever (50.0% versus 32.7%, P = 0.07), clinical sepsis (50.0% versus 38.5%, P = 0.22), and death (18.9% versus 5.8%, P = 0.05).
In our setting, a reduced intensity protocol in a low-income situation, the data warn against using ciprofloxacin prophylaxis during induction treatment. A lower nadir of neutrophil count and higher mortality were found in the ciprofloxacin group.
ciprofloxacin; prophylaxis; childhood acute lymphoblastic leukemia; randomized trial; low-income country
Acquired hemophilia A is rarely found in association with myeloproliferative neoplasms, such as the JAK2 kinase V617F mutation-positive chronic neutrophilic leukemia (CNL).
An 80-year-old Japanese male was diagnosed with acquired hemophilia A. He had compartment-like symptoms due to soft tissue hemorrhage in his left forearm and right lower extremity. A blood examination showed neutrophilia with a white blood cell count of 31,900/μL (91.9% neutrophils), an activated partial thromboplastin time of 69.0 seconds, coagulation factor VIII (FVIII) < 1.0%, and anti-FVIII inhibitor, 190 BU/mL. The bleeding episodes were controlled with intravenous activated prothrombin complex concentrate (FEIBA®) followed by recombinant factor VIIa (NovoSeven®). In addition, oral prednisolone (maximum dose, 30 mg/day) plus four doses of rituximab effectively suppressed anti-FVIII inhibitor levels while simultaneously reducing the neutrophil count. CNL with the JAK2 kinase V617F mutation was identified as the underlying disease.
This report describes the effectiveness of a combination of prednisolone and rituximab in managing acquired hemophilia A in an elderly man with a rare case of JAK2 kinase V617F mutation-positive CNL.
acquired hemophilia A; chronic neutrophilic leukemia; JAK2 kinase; V617 mutation; rituximab
The therapeutic landscape of chronic myeloid leukemia (CML) has changed dramatically in the last decade. In particular, the availability of imatinib mesylate, a tyrosine kinase inhibitor targeting BCR-ABL, has led to profound and durable remissions in the majority of patients. However, a couple of issues have emerged and partially obscured this scenario. First, it has become clear that a significant proportion of patients either present with primary resistance to imatinib or develop secondary resistance sooner or later during treatment. Second, although the drug is generally well tolerated, a percentage of patients eventually cease treatment because of toxicity. Bearing this in mind, second-generation tyrosine kinase inhibitors have been introduced, including nilotinib. Phase I and II studies indicate remarkable activity for this compound in CML cases resistant to imatinib, including some of those carrying BCR-ABL1 mutants. More recently, two Phase II studies and a III randomized Phase clinical trial demonstrated the superiority of nilotinib compared with imatinib in terms of complete cytogenetic and major molecular responses, which are two relevant surrogate measures of long-term survival in CML. In this paper, we review the most relevant data on nilotinib as first-line treatment for CML, and discuss the rationale for its routine use, as well as some possible future perspectives for CML patients.
chronic myeloid leukemia; nilotinib; targeted therapy; BCR-ABL1
Chronic myeloid leukemia (CML) is a malignant disease that originates in the bone marrow and is designated by the presence of the Philadelphia (Ph+) chromosome, a translocation between chromosomes 9 and 22. Targeted therapy against CML commenced with the development of small-molecule tyrosine kinase inhibitors (TKIs) exerting their effect against the oncogenic breakpoint cluster region (BCR)-ABL fusion protein. Imatinib emerged as the first successful example of a TKI used for the treatment of chronic-phase CML patients and resulted in significant improvements in response rate and overall survival compared with previous treatments. However, a significant portion of patients failed to respond to the therapy and developed resistance against imatinib. Second-generation TKIs nilotinib and dasatinib were to have higher efficiency in clinical trials in imatinib- resistant or intolerant CML patients compared with imatinib. Identification of novel strategies such as dose escalation, drug combination therapy, and use of novel BCR-ABL inhibitors may eventually overcome resistance against BCR-ABL TKIs. This article reviews the history of CML, including the treatment strategies used prediscovery of TKIs and the preclinical and clinical data obtained after the use of imatinib, and the second-generation TKIs developed for the treatment of CML.
drug resistance; tyrosine kinase inhibitors; chronic myeloid leukemia; imatinib; BCR/ABL
The daily recordings of treatment by patients with congenital hemophilia with inhibitors and their caregivers were assessed as part of the Dosing Observational Study in Hemophilia (DOSE) to understand the patterns of bypassing agent use and health-related quality of life.
Frequently bleeding patients prescribed recombinant activated factor VII as first-line therapy were eligible. Participants recorded daily paper diaries for at least 90 days and until at least four bleeding episodes had occurred. Web-based entry was optional. Assessment included bleeding status, work or school day status, bleeding episode, treatment, impact on planned activities, and health-related quality of life.
Diaries were completed by 18 adults and 19 caregivers (21 children). A total of 4063 diary days and 194 bleeding episodes over 491 bleed days were recorded. A small proportion of diary days were bleed days (8.2%) or treatment days (8.2%). Half the bleed days were not planned work or school days for patients (53%) or caregivers (48%). An exact agreement was observed between electronic and paper records for 93% of the reviewed health-related quality of life measurements.
Daily diary completion by patients and caregivers is feasible and provides insight into the impact of congenital hemophilia with inhibitors on daily activities and overall quality of life. Positive participation and completion rates were supported by frequent patient contact made by independent patient support liaison personnel.
daily assessment; recombinant activated factor VII; rFVIIa; NovoSeven; quality of life
Over the last 20 years, management for thalassemia major has improved to the point where we predict that patients’ life expectancy will approach that of the normal population. These outcomes result from safer blood transfusions, the availability of three iron chelators, new imaging techniques that allow specific organ assessment of the degree of iron overload, and improvement in the treatment of hepatitis. In October 2011, the Food and Drug Administration licensed deferiprone, further increasing the available choices for iron chelation in the US. The ability to prescribe any of the three chelators as well as their combinations has led to more effective reduction of total body iron. The ability to determine the amount of iron in the liver and heart by magnetic resonance imaging allows the prescription of the most appropriate chelation regime for patients and to reconsider what our aims with respect to total body iron should be. Recent evidence from Europe has shown that by normalizing iron stores not only are new morbidities prevented but also reversal of many complications such as cardiac failure, hypothyroidism, hypogonadism, impaired glucose tolerance, and type 2 diabetes can occur, improving survival and patients’ quality of life. The most effective way to achieve normal iron stores seems to be with the combination of deferoxamine and deferiprone. Furthermore, outcomes should continue to improve in the future. Starting relative intensive chelation in younger children may prevent short stature and abnormal pubertal maturation as well as other iron-related morbidities. Also, further information should become available on the use of other combinations in chelation treatment, some of which have been used only in a very limited fashion to date. All these advances in management require absolute cooperation and understanding of parents, children, and, subsequently, the patients themselves. Only with such cooperation can normal long-term survival be achieved, as adherence to treatment is now likely the primary barrier to longevity.
thalassemia; iron overload; iron chelation therapy; magnetic resonance imaging; deferoxamine; deferiprone; deferasirox
The health of an individual is known to vary in different countries, in the same country at different times, and in the same individuals at different ages. This means that the condition of individuals must be related to or compared with reference data. Determination of a reference range for the healthy term newborn is clinically important in terms of various complete blood count parameters. The purpose of this study was to establish a local reference range for full blood count parameters in neonatal cord plasma in Hilla, Babil, Iraq.
A total of 220 mothers and their neonates were enrolled in this cross-sectional study from February 2011 to January 2012. Maternal inclusion criteria were age 15–45 years, an uneventful pregnancy, and hemoglobin ≥ 10 g. Neonatal inclusion criteria were full term (37–42 weeks) and normal birth weight. The umbilical cord was immediately clamped after delivery of the baby; 3 mL of cord blood was then taken from the umbilical vein and collected in a tube containing ethylenediamine tetra-acetic acid, its plasma was analyzed for full blood count parameters by standard Coulter gram, and the differential leukocyte count was done manually.
Mean neonatal hemoglobin was 13.88 ± 1.34 (range 11–17.3) g/dL and mean white cell count was 10.12 ± 2.8 (range 3.1–21.6) × 109/L. Mean platelet count was 267.63 ± 60.62 (range 152–472) × 109/L. No significant differences in red cell, white cell, or platelet counts were found between males and females, except for neutrophil count. The current study shows lower levels of hemoglobin, white cells, and red cells compared with other studies, and there is agreement with some studies and disagreement with others concerning platelet count.
Most results in the current study were within the reference range. The hematological reference values for Iraqi neonatal cord plasma need to be confirmed by larger numbers of blood samples and by collecting samples from different areas in Iraq.
reference range; full blood count parameters; neonatal cord plasma
Transition of care from pediatric to adult providers is an essential step in the care of young adults with sickle cell anemia. Transition programs should be developed by individual institutions to systematically enhance the transition process for their patients. Prior to transfer, patients must be educated about their disease and personal medical history and develop skill sets required to navigate the adult health care setting. The objective of this literature review is to identify key concepts associated with transition of care for patients with sickle cell anemia. First, transition programs should be developed so that education about transition can begin at an early age. The readiness of patients and families should be assessed and education tailored to meet individual patient needs. Finally, the emotions and fears about transition should be recognized and addressed prior to transition.
anemia; sickle cell; transition to adult care; health transition; adult care
Pernicious anemia (also known as Biermer’s disease) is an autoimmune atrophic gastritis, predominantly of the fundus, and is responsible for a deficiency in vitamin B12 (cobalamin) due to its malabsorption. Its prevalence is 0.1% in the general population and 1.9% in subjects over the age of 60 years. Pernicious anemia represents 20%–50% of the causes of vitamin B12 deficiency in adults. Given its polymorphism and broad spectrum of clinical manifestations, pernicious anemia is a great pretender. Its diagnosis must therefore be evoked and considered in the presence of neurological and hematological manifestations of undetermined origin. Biologically, it is characterized by the presence of anti-intrinsic factor antibodies. Treatment is based on the administration of parenteral vitamin B12, although other routes of administration (eg, oral) are currently under study. In the present update, these various aspects are discussed with special emphasis on data of interest to the clinician.
anemia; pernicious; autoimmune diseases; gastritis; atrophic; neurologic manifestations; vitamin B12 deficiency