Sideroblastic anaemia in a patient with sickle cell disease.

Sideroblastic anaemia is a rare condition. We report a unique case of concomitant sideroblastic anaemia in a patient with sickle cell disease with long-standing blood transfusion history. Due to a low prevalence of sideroblastic anaemia, the diagnosis of sideroblastic anaemia is often difficult, especially when coexisting with common types of anaemia, including sickle cell disease.

Iron-Storage Disorder Presenting as Chronic Diarrhea.

The involvement of the endocrine pancreas leading to bronze diabetes is well studied. However, little is known about the pathophysiology of iron dysregulation involving the exocrine pancreas. We present a unique association between the exocrine pancreas and iron dysregulation.

Continuous infusion factor replacement in haemophilia B during and after cardiac surgery: the better choice?

A 57-year-old man with mild haemophilia B was admitted for coronary artery bypass graft surgery. His factor IX (FIX) activity was 15% on admission. Our goal was to maintain his FIX activity at 80%-100% for post-op days (PODs) 0-3, and at 60%-80% for PODs 4-14.

Assessment of health-related quality of life among adults hospitalized with sickle cell disease vaso-occlusive crisis.

Sickle cell disease (SCD) is characterized by painful vaso-occlusive crises (VOCs). Self-reported pain intensity is often assessed with the Numeric Rating Scale (NRS), whereas newer patient-reported outcome measures (PROMs) assess multidimensional pain in SCD. We describe pain experiences among hospitalized adults with VOCs, using 2 PROMs: the Patient-Reported Outcomes Measurement Information System (PROMIS) Global Health and the Adult Sickle Cell Quality of Life Measurement System (ASCQ-Me).

Phase 1 dose-finding study of rebastinib (DCC-2036) in patients with relapsed chronic myeloid leukemia and acute myeloid leukemia.

A vailable tyrosine kinase inhibitors for chronic myeloid leukemia bind in an adenosine 5'-triphosphate-binding pocket and are affected by evolving mutations that confer resistance. Rebastinib was identified as a switch control inhibitor of BCR-ABL1 and FLT3 and may be active against resistant mutations. A Phase 1, first-in-human, single-agent study investigated rebastinib in relapsed or refractory chronic or acute myeloid leukemia.

Cost Implications of Comorbidity for Autologous Stem Cell Transplantation in Elderly Patients with Multiple Myeloma Using SEER-Medicare.

Comorbidity is more common in older patients and can increase the cost of care by increasing toxicity. Using the SEER-Medicare database from 2000 to 2007, we examined the costs and life-year benefit of Auto-HSCT for MM patients over the age of 65 by evaluating the difference over time relative to comorbidity burden. One hundred ten patients had an Auto-HSCT in the early time period (2000-2003) and 160 in the late time period (2004-2007).

Cost-Effectiveness of Autologous Hematopoietic Stem Cell Transplantation for Elderly Patients with Multiple Myeloma using the Surveillance, Epidemiology, and End Results-Medicare Database.

In the past decade, the number of autologous hematopoietic stem cell transplants (Auto HSCT) for older patients with multiple myeloma (MM) has increased dramatically, as has the cost of transplantation. The cost-effectiveness of this modality in patients over age 65 is unclear. Using the Surveillance, Epidemiology, and End Results-Medicare database to create a propensity-score matched sample of patients over age 65 between 2000 and 2007, we compared the survival and cost for those who received Auto HSCT to those who did not undergo transplantation but survived at least 6 months after diagnosis, and we calculated an incremental cost-effectiveness ratio (ICER).

Sickle cell disease due to compound heterozygosity for Hb S and a novel 7.7-kb beta-globin gene deletion.

A young woman originally from Cape Verde islands presented with mild sickle cell disease. Her blood counts and hemoglobin analysis results initially suggested that she might be either homozygous for the sickle cell hemoglobin (Hb S) with concomitant alpha-thalassemia, or compound heterozygous for Hb S and beta0-thalassemia, deletional deltabeta-thalassemia or hereditary persistence of fetal hemoglobin (HPFH). We utilized a novel polymerase chain reaction (PCR)-based screening technique and found a hitherto unrecognized 7.