ASH: Sotatercept Boosts Hemoglobin in MPN-Associated Myelofibrosis

The investigational activin receptor IIA ligand trap sotatercept safely increases hemoglobin levels in patients with myeloproliferative neoplasm (MPN)-associated myelofibrosis, both when used alone and in combination with ruxolitinib (Jakafi).

A phase II investigator-initiated trial of sotatercept in this population showed responses in 10 of 28 evaluable patients, said Prithviraj Bose, MD, of updated findings presented at the 2017 ASH Annual Meeting.

Anemia is present in about one-third of patients with myelofibrosis at diagnosis and eventually develops in all patients. Only 20% to 30% of patients respond to current therapy, such as danazol and erythroid-stimulating agents.

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ASH: The Reveal Study Results for PV Patients Treated with Hydroxyurea

Examining the Treatment Patterns and Blood Counts Among Patients with Polycythemia Vera Treated with Hydroxyurea in the United States: An Analysis from the Reveal Study


Introduction: Polycythemia vera (PV) is associated with erythrocytosis (with or without thrombocytosis/leukocytosis), increased risk of thrombosis, and symptoms including fatigue, early satiety, and abdominal discomfort. Hydroxyurea (HU) is a common cytoreductive strategy used to control blood counts and splenomegaly in patients with PV. Although blood counts in many patients with PV are effectively controlled with HU, a proportion of patients fail to achieve controlled blood counts. The European LeukemiaNet (ELN) has defined response criteria for blood count control as well as HU resistance and intolerance in patients with PV; 1 criterion for HU resistance includes a lack of blood count control at a minimum dose of ≥ 2 g/day HU for ≥ 3 months.

The REVEAL observational study (, NCT02252159) is being conducted to describe contemporary demographics, burden of disease, clinical management, patient-reported outcomes, and healthcare resource utilization among patients with PV in the United States. The objective of this analysis is to describe HU treatment patterns, blood count control, and HU intolerance among patients enrolled in REVEAL.

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ASH: Pegylated Interferon Alfa-2a Active in Hydroxyurea-Resistant PV and ET

Treatment with pegylated interferon alfa-2a led to objective responses in about two-thirds of patients with hydroxyurea (Hydrea)-resistant/intolerant polycythemia vera (PV) or high-risk essential thrombocytopenia (ET), a phase II study of salvage therapy showed.

At 12 months, 69% of patients with ET and 60% of those with PV had achieved objective responses. The overall response rates (ORR) included complete responses (CRs) in 43% of the ET group and in 22% of the PV group.

No new safety or toxicity signals emerged from the MPD-RC 111 study, Abdulraheem Yacoub, MD, an associate professor of medicine at the University of Kansas Medical Center, reported at the 2017 ASH Annual Meeting.

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Update on Impact BioMedicine’s Fedratinib

ASH Highlights

Many patients wondered what happened to Fedratinib, the drug Sanofi had in trials several years ago.  Amid some controversy, the drug was shelved, although many patients were experience positive responses. Fedratinib is back. It is a selective JAK2 Inhibitor, whereas existing treatments target both JAK1 & 2. Spleen reduction will likely continue to be a primary endpoint. For those patients resistant to current treatments, Fedratinib could be a potential option in the near future.

In the JAKARTA phase 3 trial, fedratinib had a ≥35% reduction in spleen volume at week 24 in 46.9% of myelofibrosis patients and a ≥50% reduction in total symptom score in 37.1% of patients. The only grade >2 TEAE seen notably more often in 400mg fedratinib vs. placebo by cycle 6 placebo cross-over was anemia (common to all Jak2 inhibitors). We know there is a significant unmet need in the MF patient population and we’re working diligently to get fedratinib to market to address this unmet need.  In JAKARTA 2 fedratinib had a ≥35% reduction in spleen volume at week 24 in 55.4% of myelofibrosis patients that were previously intolerant or resistant to ruxolitinib treatment.

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ASH Highlights- Foggy Brain: Side Effect or Vitamin Deficiency?

Marina Sampanes Peed

Many of us in the MPN world have experienced:

  • read a page and can’t recall what you just read
  • forget events, tasks, or activities that you used to remember
  • difficulty in thinking clearly

We jokingly call it “chemo brain” and chalk it up to various medications. But what if it’s caused by something else?

According to Catriona Jamieson, MD, PhD, University of California San Diego Cancer Center, it is well-documented that deficiency of thiamine pyrophosphate deficiency (vitamin B-1) causes what we refer to as “chemo brain” or “foggy brain.” Extreme cases of thiamine deficiency can cause Wernicke’s encephalopathy (biochemical lesions on the brain), that affect vision, confusion, and memory.

Causes of thiamine (Vitamin B-1) deficiency

The body doesn’t produce essential vitamins; they must be ingested through eating healthy foods to maintain normal health.

Cancer cells metabolize faster than normal cells and they draw upon the body’s nutrient resources including glucose and micronutrients such as vitamins niacin, folic acid, pantothenic acid, pyridoxine, biotin riboflavin, and thiamine (Vitamin B1).

It’s quite common for patients to forget their nutritional needs when appetite is suppressed due to splenomegaly or certain medications.

Just one of these factors is enough to cause foggy thinking. When experiencing disease progression, the cancer cells are dominating the use critical nutrients.

Weigh the Risks

An association between cancer and low thiamine levels is demonstrated in several reports. The use of supplemental vitamins to modulate cancer rates has been promoted and discounted for years.

Some argue thiamine supplements may contribute to tumor cell survival, proliferation, and chemotherapy resistance.   Other studies suggest that very high dose thiamine produces growth inhibition of malignant cells.

What’s the answer?

Like many other elements, there is no single one size fits all answer. Talk with your hematologist about possible thiamine deficiency. If it’s low, proper dosage of Vitamin B-1 might clear the fog. Vitamins are medicine for your body, so It is important to make any changes with the knowledge and guidance of your physician.For more information check out these articles:

The Role of Thiamine in Cancer: Possible Genetic and Cellular Signaling Mechanisms

Linking Vitamin B1 with Cancer Cell Metabolism


MPN Expert Ruben Mesa on the Anemia-Based Data at ASH

At the 59th American Society of Hematology (ASH) Meeting and Exposition, Rare Disease Report caught up with Ruben Mesa, M.D., Director of the University of Texas Health Cancer Center.

In this video, he discusses the common problem of anemia that can come with myeloproliferative neoplasm (MPN)-associated myelofibrosis, how some of the medications administered can induce it, and the data pertaining to it that was presented at ASH.

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Ruxolitinib Beneficial in Hydroxyurea Resistant/Intolerant Polycythemia Vera

Data presented at the 2017 American Society of Hematology Annual Meeting (ASH 2017) are confirming previous studies suggesting that patients with polycythemia vera (PV) who fail hydroxyurea and are treated with ruxolitinib may have better hematocrit control. Researchers reported that patients treated with ruxolitinib appear to experience benefits in terms of hematocrit control, hematologic remission, and reduction in spleen size.

The RESPONSE study demonstrated that this potent JAK1/2 inhibitor results in superior response rates compared with best available therapy in controlling hematocrit and improving splenomegaly and symptoms in patients with PV whose disease was inadequately controlled with hydroxyurea. Following initial trials, an expanded-access phase 3b study was conducted looking at ruxolitinib in patients who were hydroxyurea resistant/intolerant. The study included patients who had no other treatment options available and were not eligible for any ongoing clinical trial.

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Treatment Decision Support Tool Analysis Suggests Many Clinicians Suboptimally Manage Patients With PV and MF

Many clinicians suboptimally manage patients with polycythemia vera (PV) and myelofibrosis (MF), according to an analysis of data captured from an online tool developed by Clinical Care Options (CCO) in collaboration with a panel of PV and MF experts. The analysis was presented by Ryan Topping, PhD, at the 59th ASH Annual Meeting & Exposition in Atlanta, Georgia.

Treatment for PV and MF continues to be refined, resulting in educational gaps for many healthcare providers (HCPs) who manage these conditions. In collaboration with a panel of PV and MF experts, CCO developed an online treatment decision support tool to assist HCPs in making optimal management choices for patients with PV and MF. HCPs were asked to enter patient case details into the decision support tool and were surveyed on their intended treatment for the case. They were then provided with management choices for that case from the expert panel.

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ASH Abstract: Effective Treatment of Low-Doses Decitabine in MPNs with JAK2/V617F

Primary myeofibrosis (PMF) is one of the Ph negative myeloproliferative neoplasms (MPN). The mainly clinical features are obviously physical symptoms and symptomatic splenomegaly. It may be converse to leukemia and has shortened life expectancy. Nowadays, the therapy of PMF is aimed at maintaining comfort and there was no effective treatments. PMF complicated with myelodysplastic syndrome (MDS), which is named as MDS/MPN-u, is a rare case, and the treatments are confused. In this study, we want to discuss an effective treatment in MDS/MPN via a case therapy and literature review.

A 55-year-old woman presented with fatigue and chest distress for one month was admitted in our hospital. Physical examinations showed anemic appearance and splenomegaly which was four fingers under lib. A routine blood count test showed pancytopenia. A bone marrow examination showed dysplasia and fibrous tissue proliferation. The JAK2/V617F mutation was positive and the expression was 60.63%. The chromosome karyotype showed 47, XX, t (1; 20) (p11.2; q11.2), +9,-13, +21. She was diagnosed as PMF complicated with MDS (MDS/MPN) according to WHO 2016 version of hematologic neoplasms classification. She received thalidomide 100mg daily therapy combined with prednisone.

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