BARRY S. SKIKNE, M.D.

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This study tested oral azacitidine (CC-486) as maintenance therapy after stem cell transplant in patients with acute myeloid leukemia or myelodysplastic syndromes. Relapse rates were low — especially in patients receiving the 14-day monthly schedule (13%) — and one-year survival was around 80–86%. The treatment was well tolerated with low rates of serious graft-versus-host disease, suggesting oral maintenance after transplant may help prevent relapse.

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This trial tested a 21-day-per-month dosing schedule of oral azacitidine (CC-486) in patients with myelodysplastic syndromes, chronic myelomonocytic leukemia, or acute myeloid leukemia. The drug produced responses in roughly a third of patients and freed some from needing blood transfusions, even in patients who had previously failed injectable treatments. The main side effects were low white blood cell counts and gastrointestinal symptoms, and the extended schedule was generally manageable.

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Researchers investigated whether oral azacitidine (CC-486) is safe and effective in myelodysplastic syndrome patients who already have low platelet counts. Response rates were similar regardless of starting platelet levels (38% in the low-platelet group vs. 46% in the higher-platelet group), and serious bleeding was rare. The drug was generally tolerable even in patients with the greatest bleeding risk, supporting its use across platelet count categories.

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This study examined how chromosomal abnormalities and gene mutations affect survival in older acute myeloid leukemia patients treated with azacitidine versus standard chemotherapy. Patients with the most dangerous chromosomal patterns had 31–46% lower risk of death with azacitidine compared to conventional treatment. Certain gene mutations — including FLT3 and TET2 — were linked to worse outcomes specifically in azacitidine-treated patients, pointing to areas where treatment may need to be refined.

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This analysis asked whether patients with myelodysplastic syndromes who have an underactive bone marrow (hypocellular marrow) respond differently to azacitidine than those with normal marrow cellularity. Azacitidine improved survival in both groups equally, and side effects were similar regardless of bone marrow status. Doctors can treat patients with hypocellular marrow the same way as others with this condition.

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This study investigated why patients with chronic hepatitis C frequently develop low platelet counts. The main driver turned out to be an enlarged spleen trapping and destroying platelets, rather than the liver producing too little of the hormone that stimulates platelet production. Understanding this mechanism is important for choosing the right treatment approach in these patients.

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This multicenter trial evaluated a blood test — the soluble transferrin receptor (sTfR) and a combined index (sTfR/log ferritin) — for telling apart iron deficiency anemia from anemia caused by chronic disease. Using all three markers together (ferritin, sTfR, and the index) correctly identified iron deficiency in 92% of cases, more than double the 41% detected by ferritin alone. This combination approach is particularly valuable when standard tests give ambiguous results in patients with both conditions at once.

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This case report describes a patient with chronic myeloid leukemia who developed a large, locally aggressive genital wart (giant condylomatosis) caused by HPV after a bone marrow transplant suppressed their immune system. Surgical removal combined with topical imiquimod cream resolved the tumor. The case highlights the need for aggressive treatment and close monitoring of HPV-related lesions in immunosuppressed transplant recipients.

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This review explains how measuring the serum transferrin receptor (sTfR) in blood works as a clinical tool for assessing iron status. The sTfR rises when the body is iron-deficient — unlike ferritin, it is not distorted by inflammation — and also climbs when red blood cell production is increased for any reason. Combining sTfR with ferritin allows doctors to track iron status across the full range from iron overload to severe deficiency and to distinguish iron deficiency anemia from anemia of chronic inflammation.

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This 20-year single-center review asked whether autologous stem cell transplant can cure some patients with intermediate- or high-risk acute myeloid leukemia who have no matched donor. No relapses occurred after 2.2 years post-transplant, and the survival curve appeared to plateau, suggesting that a small fraction of patients may be cured. Autologous transplant appears to be a reasonable alternative when allogeneic transplant is not an option.

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This Phase III trial compared plasma exchange using standard fresh-frozen plasma versus photochemically treated plasma (to inactivate potential pathogens) in patients with the life-threatening clotting disorder thrombotic thrombocytopenic purpura. Remission rates and time to response were nearly identical between the two treatments (82% vs. 89%). The pathogen-reduced plasma was as safe and effective as standard plasma, supporting its use as an alternative that reduces infection risk from transfusion.

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This report describes five patients with relapsed thrombotic thrombocytopenic purpura — a dangerous clotting disorder — who were all treated with rituximab after standard plasma exchange and other therapies had failed. All five achieved complete remission within about five weeks of the first dose, and all remained in remission for 10–21 months. The findings add evidence that rituximab, which targets the immune cells making disease-causing antibodies, is an effective option for relapsed disease.

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This retrospective review examined outcomes for 24 patients with relapsed or high-grade follicular lymphoma treated with autologous stem cell transplantation. Overall survival was 71.6% and disease-free survival was 40% with a median follow-up of six years, substantially better than historical outcomes with chemotherapy alone. Transplanting patients already in complete remission produced the best results, but even patients transplanted with active disease achieved meaningful responses.

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This paper describes a method for calculating total body iron stores using the ratio of serum transferrin receptor to serum ferritin, producing a single number across the full spectrum from iron surplus to iron deficit. The method was validated in healthy men and women and in pregnant women, and was used to measure iron absorption in supplementation and fortification trials in Jamaica and Vietnam. Quantifying body iron this way greatly improves the ability to detect iron deficiency and measure the effectiveness of iron intervention programs.

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This case report describes a patient who developed a fatal brain disorder (encephalopathy) three weeks after high-dose chemotherapy and stem cell transplantation for metastatic breast cancer. Brain imaging and biopsy showed damage to deep gray matter structures, a pattern different from the white matter damage more commonly described with other chemotherapy regimens. The case raises awareness that severe brain toxicity can follow this widely used treatment protocol.

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This retrospective study compared long-term outcomes in patients with severe aplastic anemia treated with either bone marrow transplantation or immunosuppressive therapy (antithymocyte globulin plus cyclosporin A) at a single center over more than 20 years. Survival was markedly better with immunosuppressive therapy (78% at five years) than with transplantation (33% at five years), even though the transplant group was younger. Long-term remissions are achievable with immunosuppressive therapy.

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This study measured how erythropoietin therapy affects the body's ability to absorb dietary and supplemental iron in kidney dialysis patients. Starting erythropoietin boosted absorption of both food iron and iron supplements by 2–4-fold, driven by the combined effect of increased red blood cell production and falling iron stores. Once patients reached a stable erythropoietin dose and blood counts had recovered, iron absorption returned to near-baseline levels.

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A woman taking methotrexate (a common arthritis drug) for five years developed abnormal lymph node swelling that initially shrank when the drug was stopped, but later came back and turned out to be Hodgkin's lymphoma, a type of blood cancer. This case shows that methotrexate can trigger lymph node problems that may look like they're going away but can actually transform into real cancer. Doctors need to keep monitoring patients who take this drug long-term, even if their swollen lymph nodes seem to improve, because cancer can develop silently without causing symptoms.

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This randomized, double-blind trial tested whether adding glutamine to nutrition support (oral or via IV) improves outcomes for bone marrow transplant patients. Glutamine supplementation did not significantly reduce hospital stays, infection rates, mucositis, or other common complications compared to placebo. The data suggested a possible reduction in the need for IV nutrition and a hint of improved long-term survival, but no clear benefit was established.

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This study tested whether iron status can be reliably measured using dried blood spots — drops of blood on filter paper, useful for large-scale field surveys. Transferrin receptor measurements worked well in dried blood spots with little interference from red blood cells, while ferritin measurements were unreliable due to contamination from hemolyzed cells. The receptor-to-ferritin ratio in dried blood spots could identify iron deficiency anemia, though it was less accurate for detecting milder deficiency without anemia.

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This study investigated which lab tests best predict whether kidney dialysis patients will respond to erythropoietin therapy and detect hidden iron deficiency during treatment. Serum transferrin receptor was useful for predicting a response when starting erythropoietin, but lost its reliability for detecting iron deficiency during ongoing treatment because the drug itself raises receptor levels by stimulating red cell production. Automated reticulocyte counts provided a complementary measure for monitoring response to dose increases.

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This animal study examined whether lung immune cells (alveolar macrophages) accumulate iron after inhaling iron-rich dust or an inflammatory mineral dust. Both iron oxide and calcium tungstate dusts caused macrophages to progressively store more iron and ferritin over the weeks following exposure. These findings suggest that macrophages sequester iron in response to lung particle exposure — a process that could either limit or contribute to oxidative lung injury.

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This study examined whether iron proteins (ferritin) found in stool reflect iron absorption and overall body iron stores. Stool L-rich ferritin levels tracked closely with body iron status in healthy people and iron-deficient patients, but were unexpectedly low in hemochromatosis patients despite their iron overload. These findings help explain how the intestine handles iron and why absorption regulation fails in hemochromatosis.

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This review describes the newly identified serum transferrin receptor — a circulating fragment of the cellular iron-uptake protein — and its potential as a clinical measurement. Blood levels of this receptor rise with tissue iron deficiency and with increased red blood cell production, and combining it with serum ferritin gives a sensitive quantitative picture of iron status. The authors anticipated it would become important for diagnosing iron deficiency anemia in both clinical practice and population studies.

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This review describes the global scope of iron deficiency anemia, which affects over 500 million people, and outlines advances in measuring, preventing, and treating it. Beyond anemia itself, iron deficiency impairs brain development in young children, reduces work capacity in adults, and increases pregnancy complications. The review evaluates tools like the serum transferrin receptor for distinguishing true iron deficiency from other anemias, and discusses fortification strategies targeted to high-risk groups.

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This study measured how much iron and ferritin lung immune cells (alveolar macrophages) release in cigarette smokers compared to non-smokers. Heavy smokers' macrophages released iron and ferritin into the airways at rates 2–10 times higher than non-smokers, with the most iron-loaded cells releasing the greatest proportion. Released iron in lung fluid could generate cell-damaging free radicals, potentially contributing to lung injury in smokers.

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This review summarizes the clinical utility of the serum transferrin receptor, a blood test that reflects the body's demand for iron at the cellular level. Unlike ferritin, it rises with iron deficiency regardless of inflammation, and also rises when the bone marrow is producing more red blood cells than usual. Combined with ferritin measurements, it allows quantitative assessment of iron status and can replace more invasive bone marrow examination in many situations.

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This study measured serum transferrin receptor levels in children with sickle cell disease to see if it could track the severity of their expanded red blood cell production. Receptor levels were substantially higher in sickle cell patients than in those with milder sickle-hemoglobin C disease or healthy controls, and rose with age and with reticulocyte counts. The findings support using serum transferrin receptor as a non-invasive marker of erythropoietic burden in sickle cell disease.

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This study searched for and found a circulating soluble form of the erythropoietin receptor — the protein that tells bone marrow to make more red blood cells — in the blood of patients with high levels of red blood cell production. The receptor fragment appeared in conditions with expanded red cell production such as sickle cell disease and thalassemia, and in patients given erythropoietin, then disappeared after marrow destruction and reappeared with engraftment. Detecting this circulating fragment offers a non-invasive way to monitor erythropoietic activity.

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This case series examined whether abnormalities in the blood's ability to dissolve clots (fibrinolysis) are present in patients with avascular necrosis of the hip, a condition where bone tissue dies from loss of blood supply. All five patients had measurable defects in fibrinolysis, most commonly elevated levels of a clot-preservation protein (PAI-1), along with high triglycerides. The findings suggest that impaired clot breakdown may contribute to the vascular blockage that causes avascular necrosis, and that fibrinolytic testing and a lipid profile are warranted in these patients.

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This study measured iron, ferritin, and two ferritin subtypes in lung immune cells (alveolar macrophages) from non-smokers, smokers with normal lung function, and smokers with chronic airflow obstruction. Smokers had 6–7 times more L-ferritin in their macrophages than non-smokers, and smokers with airflow obstruction had even higher levels, while the protective H-ferritin subtype did not increase proportionally. The disproportionate accumulation of L-ferritin — which releases iron more readily — may help explain why heavy smokers are more vulnerable to oxidative lung damage.

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This study tested whether serum transferrin receptor levels can reliably separate iron deficiency anemia from anemia of chronic inflammation — a common diagnostic challenge — and from anemia in liver disease. Receptor levels were elevated only in iron deficiency and were normal in chronic inflammatory disease and most liver disease patients, unlike ferritin which is unreliable in those conditions. The serum transferrin receptor is a reliable marker of iron deficiency that is not distorted by inflammation or liver dysfunction.

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This study measured how erythropoietin injections affect iron absorption from food and supplements in healthy volunteers. Erythropoietin treatment caused a 5-fold increase in dietary nonheme iron absorption and roughly a 3-fold increase in heme iron absorption, driven by both falling iron stores and increased demand from the stimulated bone marrow. Even after accounting for the drop in iron stores, erythropoiesis itself contributed about a 2.5–3-fold boost in absorption.

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This study examined serum transferrin receptor levels in patients with megaloblastic anemia caused by vitamin B12 deficiency, a condition where the bone marrow tries but fails to make functional red cells. Receptor levels were elevated only in the most severely anemic patients, and paradoxically rose further after B12 treatment as effective red cell production replaced the failed attempts. The pattern confirms that the receptor reflects actual functional red cell production, and its changes can help monitor recovery.

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This case series describes five patients with severe aplastic anemia treated sequentially with antithymocyte globulin first, then cyclosporin when initial treatment did not produce a response. All five patients improved: four became transfusion-independent and one had partial improvement, with responses appearing within four months of starting cyclosporin. Sequential immunosuppression appears effective for aplastic anemia and warrants further evaluation.

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This study tested whether serum transferrin receptor measurements are useful for detecting iron deficiency during pregnancy. Receptor levels in third-trimester women were no higher than in non-pregnant women, so pregnancy itself does not elevate the test, and elevated levels reliably indicated iron depletion. Combining receptor and ferritin measurements allowed assessment of the full spectrum of iron status in pregnant women.

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This controlled phlebotomy study in healthy volunteers established the serum transferrin receptor as a quantitative measure of iron deficiency by systematically draining iron stores through repeated blood draws. Receptor levels stayed normal while iron stores were being depleted but rose steadily once stores were exhausted and the body started pulling iron from tissues. The serum transferrin receptor proved more sensitive and reliable than other standard tests for detecting early, mild iron deficiency.

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This study evaluated a gastric delivery system (GDS) — a capsule that releases iron slowly in the stomach — as an alternative to standard iron tablets. The GDS provided three times better iron absorption than conventional ferrous sulphate, and in a double-blind trial of 200 women, the GDS produced no more gastrointestinal side effects than placebo while standard tablets caused significantly more nausea and loss of appetite. A single daily GDS capsule delivers as much absorbed iron as three conventional tablets taken throughout the day, with far fewer side effects.

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This study measured serum transferrin receptor levels across a range of blood cancers to see whether the test could reflect disease activity. Levels were significantly elevated in myeloproliferative disorders and chronic lymphocytic leukemia, where receptor levels tracked disease stage, but were normal in lymphoma, myeloma, and most other cancers. In chronic lymphocytic leukemia the test may serve as a marker of disease burden, while in other cancers it mainly reflects red blood cell production activity.

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This study examined whether serum transferrin receptor levels — which reflect the total amount of cellular iron-uptake protein in the body — predict how much iron a person absorbs from food or supplements. Receptor levels correlated far less with iron absorption than ferritin did, and the correlation disappeared entirely in people who were not iron deficient. In healthy individuals, the size of iron stores measured by ferritin is the main regulator of absorption, not the amount of transferrin receptor.

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This study determined the molecular structure of the serum transferrin receptor — the circulating form of the cellular iron-uptake protein. Purification and protein sequencing showed it is a truncated fragment missing the first 100 amino acids (the parts normally anchored inside and across the cell membrane), and it circulates bound to transferrin. This structural identification confirmed the serum receptor is a genuine shed form of the tissue receptor, establishing the foundation for interpreting blood-level measurements.

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This study examined the iron-handling proteins in the lining of the small intestine in patients with hereditary hemochromatosis compared to healthy controls. In hemochromatosis, mucosal ferritin — which normally rises with iron loading to block further absorption — failed to increase appropriately despite massive iron overload, while the iron-transport protein transferrin was significantly lower. The data point to a defect in the intestinal cell's normal iron-sensing mechanism as central to the disease, though it remains unclear whether this is cause or consequence of the abnormal absorption.

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This study focused on a new test for measuring a protein called transferrin receptor in the blood. Researchers found that healthy individuals had an average of 5.63 mg/L of this protein, while levels dropped to about 2.58 mg/L in patients with severe blood disorders like aplastic anemia and rose to around 33.1 mg/L in those with hemolytic anemia or iron deficiency. This new testing method is much more sensitive than older techniques and helps doctors better understand and diagnose different blood conditions. Who this helps: Patients with anemia and their doctors.

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This study focused on how to better identify and measure iron deficiency in patients. Researchers found that serum ferritin, which indicates body iron levels, is the best and most cost-effective way to screen for iron deficiency, allowing for easier diagnosis compared to more invasive tests like bone marrow examinations. Additionally, measuring the serum transferrin receptor may provide even more accurate information about tissue iron supply. Who this helps: This helps patients suffering from iron deficiency and doctors diagnosing their condition.

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This study measured absorption of both heme and nonheme iron from a hamburger meal in patients with hereditary hemochromatosis, their heterozygous relatives, and healthy controls. Hemochromatosis patients absorbed far more iron than predicted by their iron stores, confirming a fundamental defect in absorption control, and they were especially susceptible to loading from heme iron. Even heterozygous carriers showed a detectable absorption abnormality when the test meal contained a large, highly absorbable iron supplement.