Chronic illness is a lonely thing

"For the past year I religiously text my husband, Shea, every Friday afternoon. There’s nothing unusual in that, but the weekly message contains just a number, no words. Triple digits and we’re happy; single digits and it’s time to get family in to mind the children and pack a bag for hospital.

The number I text is my platelet count. I am one of approximately 1,000 people in Ireland with ITP (immune thrombocytopenia), a rare blood disorder in which the immune system mistakenly attacks its own platelets. It can occur in pregnancy, follow a virus, vaccination or certain medications, but for most people the cause is unknown."

-By Karen Tomkins for The Irish Times. Image courtesy of Karen Tomkins and The Irish Times.

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Lessons from vaccine-induced immune thrombotic thrombocytopenia

Adenoviral vector vaccines are effective against SARS-CoV-2 but have been associated with a rare side effect termed vaccine-induced immune thrombotic thrombocytopenia (VITT). Here, we discuss our understanding of how vaccine-induced antibodies to platelet factor 4 (PF4) form immune complexes that activate platelets and trigger the thrombotic events seen in VITT.

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SARS-CoV-2 Vaccination and Immune Thrombocytopenia in de novo and pre-existing ITP patients

Key Points

  • SARS-CoV-2 vaccines are generally safe in patients with pre-existing ITP but thrombocytopenia exacerbation may occur and requires monitoring
  • Splenectomy and past use of 5 or more therapies predict higher risk of worsening thrombocytopenia in ITP patients post-SARS-CoV-2 vaccine

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Mum's horror condition that put her at risk of fatal bleeding while pregnant

“When I was first diagnosed, they said my platelet levels would continue to drop and there was a risk of internal bleeding which can ultimately be life-threatening,” she said.

“This is why supporting plasma donation is such a big thing for me. The only reason I was able to continue having children is they knew immunoglobulin worked for me and it would help me boost my platelet levels straight away."

- By Paige Oldfield. Image courtesy NHS.

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Boy diagnosed with bleeding disorder after mum notices red dots on his ear

“When Hudson was six months old, he picked up a few infections, but our GP wasn’t concerned as he always bounced back. But a month later, I noticed tiny dots on his ears that spread over his body, and then bruises on his legs. I felt panicked. Could this be Meningococcal?

A GP thought it was Idiopathic Thrombocytopenic Purpura (ITP) and while he was arranging blood tests, I noticed Hudson’s nostrils were covered in blood! When that happened, the doctor’s mood shifted to a sense of real urgency, and we were sent straight to the hospital."

- By Carrol Baker, Image courtesy of the family.

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A Place in the Sun's Laura Hamilton emotional as guest's sister dies from disorder she has

“She, like myself, was an ITP sufferer, which is shortened for Immune thrombocytopenia, which is a disease which means your platelet count can be very low."

“Tragically Hazel’s platelet count was so low that she had a bleed on the brain.”

Laura went on to explain how this is a condition she has had herself since giving birth to her daughter.

- By Fay Watcon for Image courtesy Instagram/Laura Hamilton.

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McMaster University logo

McMaster team closes in on cause of rare blood clots related to AstraZeneca COVID-19 vaccine

"Canadian researchers have established the most definitive link yet between the Oxford-AstraZeneca COVID-19 vaccine and a rare blood clotting syndrome that shattered public confidence in the vaccine earlier this year.

The work, conducted by a team at McMaster University, has yielded a more efficient way to test for the syndrome and also lays out the underlying mechanism that is producing blood clots in a small fraction of vaccinated individuals."

- By Ivan Semeniuk, Science Reporter, The Globe and Mail

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Update - Health Canada issues label change on the AstraZeneca and COVISHIELD COVID-19 vaccines

OTTAWA, ON, June 29, 2021

Products: AstraZeneca and COVISHIELD COVID-19 vaccines

Issue: Health Canada is updating the product monograph—or label—for the AstraZeneca and COVISHIELD COVID-19/ vaccines to add capillary leak syndrome as a potential side effect, with a warning for patients with a history of capillary leak syndrome to not get the AstraZeneca or COVISHIELD COVID-19 vaccine. The label has also been updated with information about very rare events of blood clots associated with low levels of platelets following immunization.
What to do: Talk to your healthcare professional if you have any questions about the AstraZeneca and COVISHIELD COVID-19 vaccine. If you have previously had capillary leak syndrome, talk to your healthcare professional about your COVID-19 vaccine options. Seek immediate medical attention if you experience symptoms of capillary leak syndrome which would include rapid swelling of the arms and legs and sudden weight gain, which can be accompanied by feeling faint. In addition, seek prompt medical attention if you experience any symptoms that could be thrombosis with thrombocytopenia following vaccination.

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Baby's hand holding finger

Lucknow woman delivers a healthy baby, winning against COVID & a rare immune disorder

"Affected by 'Idiopathic Thrombocytopenic Purpura' (ITP), Deepika Tiwari was in a critical stage at the time of her delivery. This disease annihilates the blood platelets, thereby causing internal bleeding from the skin. Despite the uncertainties, the care and treatment by KGMU doctors aided the patient with a protected procedure.

After delivering the baby through C-section surgery, the mother and the child were both stable and healthy and they have been discharged now. Shailendra Tiwari, the father of the newborn was overjoyed to hear the good news. He recounted that tears rolled out of his eyes when he learned that the mother-daughter duo was fine and stable."

- By Ankur Verma,

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Blood platelets falling in Covid-19 cases

"Healthcare experts treating COVID-19 have revealed that they have identified several cases wherein blood platelet count was low."

- By U Sudhakar Reddy / TNN

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Meet the Canadian doctors working to identify and treat the rare blood clots linked to COVID-19 vaccines, including PDSA Medical Advisor Donald Arnold, MD

"For decades, McMaster University in Hamilton, Ont., has been a hub for research on blood and its diseases — known as hematology — but in recent weeks it has taken on an even more prominent role in the field: working to identify the rare blood-clotting syndrome linked to certain COVID-19 vaccines.

The lab, a small space on the third floor of the university hospital, is the only one in Canada with the equipment and expertise to test for the syndrome, known as vaccine-induced prothrombotic immune thrombocytopenia, or VIPIT."

- From  Image copyright and courtesy of McMaster University.

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Blood plasma illustration

Blood plasma donations for manufacture of medicines to begin after ban lifted

"Donations of blood plasma in England for use in the manufacture of “lifesaving” medicines are to begin after the lifting of a ban lasting more than two decades.

For an initial three months from Wednesday, donations will be taken at 14 centres across the country.

These will be used to make antibody-based medicines, called immunoglobulins, for people with rare immune diseases."

- By Tom Pilgrim from the Evening Standard

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Vaccine injection

What is thrombocytopenia, the rare blood condition possibly linked to the AstraZeneca vaccine?

"The federal government has asked Australia’s medical and vaccine regulators to urgently consider the European Medicines Agency’s finding of a possible link between the Oxford/AstraZeneca COVID vaccine and rare blood clots.

This follows reports over recent weeks of blood clots in a small number of people around the world who had received the AstraZeneca vaccine, including 1 man who was hospitalized in Melbourne.

Scientists have termed the condition “vaccine induced prothrombotic immune thrombocytopenia” (VIPIT). But what does this actually mean, how significant is the risk, and what are the implications for Australia’s vaccine rollout — which is currently relying predominantly on the AstraZeneca jab?"

- From ABS/CBN News, The Conversation/Reuters

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Australia's Coronacast - Here's what we know about the blood clots reports

Australian health authorities and regulators have assured the public that there is currently no evidence to suggest the AstraZeneca coronavirus vaccine is not safe or is causing blood clots.

It's come in response to several European countries pausing the AstraZeneca vaccine rollout amid blood clot concerns.

The European Medicines Agency is currently investigating reports, and is due to report back on Thursday, and as such the Australian rollout will continue.

So on today's Coronacast, what we know and what we don't about this whole story about blood clots.

- From ABC TV (Australia)

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Kristin - Living with Immune Thrombocytopenia

Canadian ITP patient and long-time supporter of PDSA, shares her experiences living with ITP.

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Sobi logo

Doptelet® (avatrombopag) approved in the EU for treatment of ITP

Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) today announces that the European Commission (EC) has approved an extension of the indication for Doptelet (avatrombopag) to include the treatment of primary chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments (e.g. corticosteroids, immunoglobulins) in all European Union (EU) member states.

- From Sobi press release

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Rigel logo

Rigel and Medison Announce Health Canada Approval of TAVALISSE®, an Oral Medication for the Treatment of Adults with Chronic ITP

Rigel Pharmaceuticals, Inc. and Medison Pharma today announced that Health Canada has approved the new drug submission (NDS) for TAVALISSE® (fostamatinib disodium hexahydrate) for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to other treatments.

"This approval of TAVALISSE provides ITP patients and physicians in Canada with a new oral treatment option, the only therapy to address the underlying platelet destruction that causes ITP," said Raul Rodriguez, Rigel's president and CEO. "With Medison as our collaborative partner, we believe TAVALISSE is well positioned for commercial success in the Canadian market."

In October 2019, Rigel entered into exclusive license agreements with Medison to commercialize TAVALISSE in Canada and Israel. With the approval from Health Canada, Medison intends to launch TAVALISSE in Canada in Q1 2021. In Israel, a decision on the new drug application (NDA) is anticipated during Q2 2021.

- From Rigel Press Release

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Sobi launch Doptelet® (avatrombopag) in Europe

Sobi™ Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO:SOBI) announced (11/04/20) the commercial launch of Doptelet® (avatrombopag) in Europe, with the United Kingdom as the first country for launch. Doptelet is a thrombopoietin receptor agonist (TPO-RA) approved for the treatment of severe thrombocytopenia in adult patients with chronic liver disease (CLD) who are scheduled to undergo an invasive procedure.

- From Sobi Press Release

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European Haematology Association (EHA) Appointment

We are extremely pleased and proud to announce the appointment of the ITP Support Association Trustee Mr Derek Elston to the European Haematology Associations, European Affairs Committee as the second patient representative from the Patient Organisation Workgroup. This is a three year appointment. The committee comprises numerous eminent haematologists from all over Europe and is chaired by the President and Vice President of EHA.

- From ITP Support Association Press Release


Hematology Society of Taiwan event raises awareness about ITP

Pop singer Selina Jen, center, and a group of medical doctors make the “love” sign at a news conference in Taipei yesterday to encourage people with immune thrombocytopenia to seek medical treatment.

ITP Patient Association chairperson Chen Chia-mei (陳佳美) said that people with the disorder also often experience social or work-related problems due to their condition, so they organized the walking event encourage patients to be brave and join in outdoor events with other people.

- By Lee I-chia / Staff reporter, Taipei Times. Photo courtesy of the ITP Patient Association

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Laura Hamilton

UK TV presenter Laura Hamilton diagnosed with bleeding disorder

"Laura Hamilton was diagnosed with a bleeding disorder after finding huge bruises.

The 38-year-old TV presenter – who has kids Rocco, five, and Tahlia, four, with husband Alex – was suddenly covered in huge bruises after welcoming her second child, which led to a terrifying diagnosis.

She told the Daily Mirror newspaper: "It was terrifying. I had been on quite a strict diet. I'd cut out sugar and carbs and I was the slimmest I'd been. I was exercising loads and felt in really good shape.

"When Tahlia was about seven months old, I was due to drive to Portugal to film a fitness app, but a few days before I was due to go I started noticing all this bruising coming out on my legs."

Eventually, her mother-in-law suggested she should see a doctor, because she suspected the bruising could be caused by a blood disorder."

- From  Photo courtesy

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Rigel Announces Availability of TAVLESSE® (Fostamatinib Disodium Hexahydrate) in Europe

Rigel Pharmaceuticals, Inc. announced that Grifols S.A., its collaborative partner in Europe, has launched TAVLESSE® in Germany and the United Kingdom. It was approved by the European Commission in January 2020 for the treatment of chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments. TAVLESSE, which is marketed in the U.S. under the brand name TAVALISSE®, is an oral spleen tyrosine kinase (SYK) inhibitor that targets the underlying autoimmune cause of ITP by impeding platelet destruction.

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ITP Support Association - ITP and Covid-19 Webinar

On Thursday, April 16th, Mervyn Morgan, CEO, Prof. Adrian Newland, CBE and Rhonda Anderson from the ITP Support Association facilitated questions to a team of world renowned ITP experts - Dr. Drew Provan, Dr. Sue Pavord and Dr Quentin Hill.

Watch Webinar Recording

Facts over Fear: COVID-19 & ITP Virtual Town Hall Meeting with Global ITP Experts

This webinar was broadcast live on April 3, 2020. Over 700 individuals registered from 44 Countries, 48 U.S. States, and 7 Canadian Provinces.


Grifols to launch TAVLESSE® in Europe and Turkey to continue reinforcing its commercial strategy and commitment to patients

Grifols announced that the European Commission has approved US-based Rigel Pharmaceuticals’s TAVLESSE® (fostamatinib) for the treatment of chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments.

Grifols has exclusive rights to fostamatinib in chronic ITP, as well as any potential future indications like autoimmune hemolytic anemia (AIHA), and IgA nephropathy (IgAN), in Europe and Turkey.

- From Grifols press release

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Tavalisse Bottles

EMA Panel Backs Fostamatinib (Tavalisse) for Chronic ITP

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval of fostamatinib disodium hexahydrate (Tavalisse, Rigel Pharmaceuticals, Inc) to treat adults with chronic immune thrombocytopenia (ITP) who fail other treatments.

- By Megan Brooks for

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Nawaz Sharif

Pakistan's ex-Prime Minister Sharif diagnosed with immune disorder

Sharif, 69, is being treated at a hospital in Lahore, where medical tests suggest he has a low count of blood platelets.

Jailed former Pakistani Prime Minister Nawaz Sharif is suffering from a serious immune disorder, his party leaders say, accusing the government of not providing him adequate medical care.

Sharif, 69, is being treated at a government-run hospital in the eastern city of Lahore, where medical tests suggest he has a dangerously low count of blood platelets, Talal Chaudhry, a senior leader of Sharif's PML-N party, told Al Jazeera on Friday.

"It is acute immune thrombocytopenic purpura, a bleeding disorder, in which the immune system destroys platelets," the doctor told Dawn, speaking on condition of anonymity. "We are hopeful that his condition will improve in a few days."

- by Asad Hashim for Photo courtesy of Wikipedia.

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Chrystal Gellein

New hope for ITP patients dashed by high drug cost

Without access to eltrombopag, Gellein continues to take other medications and suffer from side-effects of anxiety, depression, weakness, nausea and dizziness. She is admitted to hospital several times each year for emergency treatment, which is common for patients with uncontrolled ITP.

She wants people to know the ripple effect it has caused on those around her, and desperately wants her life back.

"My life could have been so much different with these drugs," said Gellein. "Something needs to change, if not for me, for the hundreds of young people being diagnosed with ITP in Canada every year."

- By Dr. Meera Dalal for CBC News.  Photo courtesy of Chrystal Gellein.

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Group in Buenos Aires

Buenos Aires First-Ever World ITP Day a Tremendous Success!

On Saturday, October 6, 2019, PTI Argentina held the first celebration of World ITP Day in Buenos Aires, Argentina. Months of hard work with almost no funding proved a big success as people turned out to show their support and listen to the voices of ITP!

After a group walk along Buenos Aires main Avenue, handing out brochures in the street, the group shared a friendly afternoon tea with a well-known hematologist who answered questions about ITP. At the end of the day everyone left with a big smile (and a full stomach), fewer doubts about ITP and a "Mission Accomplished" feeling that will motivate them to continue advocating for and supporting ITP patients.

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2019 Global Alliance meeting

2019 Global Alliance Meeting

On Thursday, July 25th, PDSA was honored to host 18 delegates from 12 countries prior to ITP Conference 2019 for the 3rd International ITP Alliance Meeting. In addition to updates from Alliance Ambassadors from around the world, the symposium included sessions about the ITP Registry, working with industry partners, the development of and capacity building. International ITP Alliance Ambassadors also had the opportunity to meet one-on-one with ITP industry partners. Representatives from Argentina, Australia, Brazil, China, Denmark, Finland, Israel, Italy, Netherlands, New Zealand, United Kingdom and the United States attended the full-day meeting and also joined in ITP Conference 2019 sessions and festivities!

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Chronic immune thrombocytopenia in Denmark, Sweden and Norway: The Nordic Country Patient Registry for Romiplostim

The NCPRR was established in April 2009 as a cohort study of all adult cITP patients in Denmark, Sweden and Norway (combined adult population = 15∙4 million persons) with study inclusion continuing through 2019. The three countries have tax-funded health care systems. ITP patients are referred by their general practitioners to hospital-based hematological specialists. Hospitals are required to report data on all hospital visits to nationwide hospital registries. Virtually complete follow-up for hospitalizations, outpatient clinic visits, and death among ITP patients is available through the countries´ nationwide hospital and population registries, linkable through the personal identification number (PIN) assigned to all residents of each country.

- From

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Chi-Med Initiates a Phase I Trial of HMPL-523 in Patients with ITP in China

Hutchison China MediTech Limited (“Chi-Med”) has initiated a Phase I study of HMPL-523, its novel spleen tyrosine kinase (“Syk”) inhibitor, in patients with immune thrombocytopenia (“ITP”), an autoimmune disorder that can lead to increased risk of bleeding. The first ITP patient was dosed on August 12, 2019 in China.

The study is a randomized, double-blinded, placebo-controlled Phase Ib clinical trial investigating the safety, tolerability, pharmacokinetics and preliminary efficacy of HMPL-523 in adult patients with ITP. The primary endpoint is the number of patients with any adverse event.  Approximately 50 to 60 patients will be enrolled.

- From Hutchison China MediTech Limited News Release

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First Irish patient day in more than a decade for rare blood condition

On 13 April 2019, immune thrombocytopenia (ITP) patients from around Ireland, were welcomed to the ITP Support Association Patient Day in Dublin supported by Novartis. Chaired by Mr. Mervyn Morgan (Chief Executive of ITP Support Association), the meeting provided patients with an opportunity to discuss their experience of ITP with other patients and to learn about their disease from experts in the field.

Last held in Dublin in 2004, this year’s meeting featured talks from Ms. Nicola Harten (Clinical Nurse Specialist, Connolly Hospital, Dublin), Dr. Gerard Crotty (Consultant Haematologist, Midland Regional Hospitals, Tullamore, Portlaoise and Mullingar), Prof. Adrian Newland (Professor of Haematology, Barts and The London School of Medicine and Dentistry, Queen Mary University of London, UK) and Mr. Derek Elston (ITP Patient Mentor).

Additional highlights from the meeting included a Q+A session, an overview of the ITP pocket log and patient breakout groups. The meeting was attended by 55 ITP patients from across the country.

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Intas logo

Intas Becomes the First to Launch Romiplostim in India, Significantly Reducing the Treatment Cost of Chronic ITP

One of India's leading pharmaceutical companies, Intas Pharmaceuticals, takes a revolutionary step towards making therapy for chronic immune thrombocytopenia (ITP) more accessible to patients by being the first to launch Romiplostim in India under the name Romy®

Romiplostim is one of the most efficacious agents with proven long-term benefits in chronic ITP. It represents the standard of care but has been available only internationally since 2008. It is estimated that around 42,000 patients are diagnosed with ITP in India every year. Less than 10% of patients which include adults as well as children, have access to the treatment which is required to maintain adequate platelet counts for a longer duration. In ITP, long term use of drugs is required to maintain adequate platelet count.

- From Intas Pharmaceuticals Press Release

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ITP Support Association is hosting a patient day

Patients living with Immune Thrombocytopenia (ITP), and their families, are invited to attend an upcoming ITP Patient Day organised by patient group ITP Support Association on Saturday 13th of April at the Ashling Hotel, Dublin. The event, which is supported by Novartis, aims to bring together people living with ITP and their families to learn from one another and leading experts in haematology about the management of ITP in a relaxed and informal setting.

- From

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ITP Weekend Planned: London, ON Canada – April 27-28, 2019

PDSA Canada logoPDSA Canada has planned a weekend all about ITP in London, ON Canada.

On Saturday, April 27, 2019 the community will Pump It Up For Platelets!

On Sunday, April 28, 2019 the Platelet Disorder Support Association will host its 2nd Canadian ITP Regional Meeting.

Full details and registration for both events

ChinaFourth China Platelet Day on 20th, March 2019

Many gather with the power of love to improve quality of life in patients.

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Genetic breakthroughs showcased on China Platelet Day

Domestic doctors can conduct precise diagnosis on platelet diseases by identifying all the 85 diseases-related genes as well as the introduction of more targeted medicine, doctors said during China Platelet Day on Wednesday.

A charity program was also launched to support patients with immune thrombocytopenia, or ITP, a type of disease characterized by a low platelet count and increased tendency to bleed.

“ITP is the most common cause of bleeding, almost covering one-third of patients with bleeding. People with ITP can suffer bleeding under the skin, inside the brain and digestive channel and people with serious conditions can die,” said Dr Liu Peng, director of the hematology department of Shanghai’s Zhongshan Hospital.

- By Cai Wenjun, Metro Managing Editor, SHINE News

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China Investigating Reports Of HIV-Tainted Medication (IVIG)

Chinese authorities say they are investigating a batch of medication that is suspected to be tainted with HIV.

According to local media, the batch contains more than 12,000 doses of human immunoglobulin — intravenous treatments used to boost weakened immune systems.

China's National Health Commission said in a statement that on Tuesday it asked medical institutions to stop using the suspect medicine, seal it and monitor any patients who might be impacted. It launched a recall of the product, which it says was produced by Shanghai Xinxing Pharmaceutical Company.

- By Merrit Kennedy,

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Statement from PPTA - Plasma Protein Therapeutics Association

Safety & Immunoglobulin Manufacturing

Recent news reports called into question the safety of immunoglobulin therapies outside
of Europe and the U.S. While the facts in this case are not clear, PPTA can provide
assurance that plasma protein therapies manufactured by PPTA member companies are
safe. All undergo viral removal and inactivation steps during production. These steps are
effective in helping to eliminate risks from HIV, hepatitis, and other blood-borne infectious
agents, and the safety record of current therapies is excellent; there have been no viral
transmissions for nearly three decades.

PPTA member companies adhere to or exceed all requirements set out by regulatory
authorities around the world, including the U.S. Food and Drug Administration (FDA), the
European Medicines Agency (EMA), and others. Additionally, PPTA member companies
comply with a robust set of voluntary industry standards, including the International
Quality Plasma Program (IQPP) and the Quality Standards of Excellence, Assurance and
Leadership (QSEAL). Learn more about PPTA's Pathogen Safety program here.



Underwood terminal cancer patient feels 'helpless' after he was refused pain drug

He was due to receive his ninth cycle of this particular drug, but developed immune thrombocytopenic purpura (ITP), which causes low platelets in his blood. ITP is not directly related to his myeloma and it is uncommon for the two conditions to occur together.

In order to treat ITP, Ian was taken off his lenalidomide until his platelet level returned to an acceptable level, which in this case took six months. NHS policy requires a treatment break application to be submitted for patients who have had a pause in their treatment.

- By Hannah Mitchell, Eastwood & Kimberly Advertiser.  Photo courtesy of Nottingham Post

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We Live Day by Day

Cooper Higgins, 7, battling rare blood disorder known as idiopathic thrombocytopenic purpura

Cooper stopped playing hockey and had to be home-schooled because of his condition. A simple nose bleed could be fatal and even a minor bump on the head could lead to internal bleeding in the brain.

Over the next year, the family cycled through medication and treatments, trying anything they could to make a difference. But nothing worked longer than a few weeks and often the side effects were even worse.

Kelly estimates they have spent thousands of dollars on medicines. However, they finally found something that is working … at least for the time being.

- By Eric Bowling, The Westlock News.  Photo courtesy of the family.

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Paras Biopharmaceuticals Finland Oy Announces Successful Development of Biologically Active Romiplostim (N-Plate® Biosimilar) Production Tsful Development of Biologically Active Romiplostim (N-Plate® Biosimilar) Production Technologyechnology

Jan. 30, 2019 / PRZen / OULU, Finland -- PARAS BIOPHARMACEUTICALS FINLAND OY today announced the achievement of a major milestone – the successful development of biologically active Romiplostim (N-Plate® biosimilar) production technology.

Romiplostim has been produced with a proprietary and innovative continuous manufacturing process at the company's Finnish-based facility in Oulu-Finland. Romiplostim is used for the treatment of thrombocytopenia (low platelet count) in patients with chronic immune (idiopathic) thrombocytopenic purpura (ITP).

ITP is a potentially serious blood disorder characterized by increased destruction and impaired production of platelets. It can lead to extensive bruising and bleeding. Thrombocytopenia is common in cancer patients, resulting from chemotherapy or radiation treatment, in addition to the underlying disease itself. ITP leads to several complications. Low platelet count increases spontaneous bleeding, complicates surgical operations and can affect chemotherapy and radiation therapy.

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Momenta Pharmaceuticals Announces First Subject Dosed in Phase 1/2 Clinical Trial of M254, Hypersialylated Immunoglobulin G

CAMBRIDGE, Mass., Jan. 29, 2019 (GLOBE NEWSWIRE) -- Momenta Pharmaceuticals, Inc. (Nasdaq: MNTA), a biotechnology company focused on discovering and developing novel biologic therapeutics to treat rare immune-mediated diseases, today announced the dosing of the first subject in the Phase 1/2 clinical trial of M254, hypersialylated Immunoglobulin G (hsIgG). The Phase 1/2 study will enroll normal healthy volunteers and patients with Immune Thrombocytopenic Purpura (ITP). The multi-part study includes single and multiple dose parts, and a placebo-controlled, randomized double-blinded cross-over study comparing M254 to IVIg.

“We believe M254 has the potential to be a significantly better option for patients than conventional IVIg. Our aim for this study is to show clinically what we have observed in extensive preclinical models, which is that hypersialylated IgG is substantially more potent than intravenous immunoglobulin G (IVIg) in ITP and other inflammatory disorders,” said Santiago Arroyo, M.D., Ph.D., Senior Vice President of Development and Chief Medical Officer of Momenta Pharmaceuticals. “We look forward to obtaining initial clinical data in the first half of 2020.”

The four part study will evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of intravenous (IV) M254 in approximately 65 subjects, including healthy volunteers and patients with ITP. Parts A and B are double-blind, placebo-controlled, single ascending dose cohort studies in healthy volunteers and ITP patients, respectively. In Part C, ITP patients will receive M254 or IVIg in a cross-over dosing design, while in Part D, ITP patients will receive multiple doses of M254. The primary efficacy endpoint is an assessment of platelet response.

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Caroline Kruse Named President and CEO of the Platelet Disorder Support Association

The Platelet Disorder Support Association (PDSA) Board of Directors is pleased to announce that Executive Director Caroline Kruse has been named President and Chief Executive Officer. Kruse, who has been Executive Director of PDSA since January 2009, will serve in the dual role focusing on the core functions of the organization including strategy, finance, board management, fundraising, and program delivery.

Under Caroline’s dedicated leadership, PDSA has developed new relationships with key disease stakeholders and government agencies and strengthened existing relationships with partners and supporters. Her focus on mission has resulted in a greater emphasis on advocacy, expanding programs for children, teens and families affected by ITP, increasing education initiatives and advancing research to improve the quality of life for those living with immune thrombocytopenia (ITP) and other platelet disorders.

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International Alliance Meeting in Chester

It was a beautiful sunny Saturday morning when all the international participants of the ITP Convention and the Alliance meeting got together at the racecourse in a beautiful city of Chester. Most of the attendees already knew each other well and the reunion was filled with joy and happiness but there were also new faces to get familiar with which was very exciting, too. Patient group representatives outside England came from the USA, Brazil, India, Italy, the Netherlands, Norway, Denmark, Sweden and Finland.

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How patients are helping drive research and drug development

On October 15, 2018, PDSA President & CEO, Caroline Kruse joined this panel discussion moderated by Christopher P. Austin, M.D., Director, NCATS, NIH at the NORD Rare Diseases & Orphan Products Breakthrough Summit. The panel discussed innovative collaborations and how the role of patients, caregivers and patient organizations has become instrumental in helping to advance research and development of therapies.

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Rigel Receives EMA Validation of the Marketing Authorization Application for Fostamatinib Disodium Hexahydrate in Chronic ITP in Adult Patients

First SYK inhibitor for the treatment of adult chronic ITP

SOUTH SAN FRANCISCO, Calif., Oct. 11, 2018 /PRNewswire/ -- Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL) today announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for fostamatinib in adult chronic immune thrombocytopenia. The validation was received on October 4, 2018 and initiated the MAA review process. The company anticipates a decision from the Committee on Human Medicinal Products by the fourth quarter of 2019. Currently, fostamatinib is commercially available in the U.S. under the brand name TAVALISSE™ (fostamatinib disodium hexahydrate), which is the first and only SYK inhibitor indicated for the treatment of thrombocytopenia in adult patients with chronic ITP who have had an insufficient response to a previous treatment.

- From Rigel Press Release

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Five-year-old Wrexham boy with unusual and rare disease takes part in photo project to raise awareness

"Charlie Clarke and his Mum Carrie, from Wrexham, are raising awareness of ITP (Immune Thrombocytopenia) by taking part in the Rare Aware photography project by Mold-based organisation Same but Different.

"It’s important to us that there is more awareness, which is why we wanted to take part in the Rare Aware campaign.

"Knowledge is power. The more people that know about ITP and other rare conditions, the better."

- By Steve Craddock, Chief Reporter, The Leader. Photo Courtesy Ceridwen Hughes.

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International ITP Alliance Members Attend Events

Hike and Seek 2018Global ITP advocacy partners from Denmark, Finland, Netherlands, and New Zealand attended the 18th ITP Conference 2018 for the first time as the International ITP Alliance. The three-day event and scholarship opportunity presented by the Platelet Disorder Support Association (PDSA), offered attendees information on the latest advancements in the diagnosis and treatment of ITP, small group sessions where patients shared their stories, an educational dinner program, a fun-filled hike and seek event and PDSA’s 20th Anniversary celebration at the legendary Rock and Roll Hall of Fame.

ITP Alliance at the 23rd Congress of European Hematology Association

EHA 2018International ITP Alliance members from Denmark, Italy, Sweden and the United States participated in the 23rd Congress of the European Hematology Association (EHA) in Stockholm, Sweden on June 14-17, 2018. It was the largest EHA Congress held to date with more than 11,000 participants from around the world. The program covered every subspecialty in hematology and attendees networked with worldwide experts. This year’s educational and scientific program highlighted state-of-the-art clinical practice, the latest findings in hematology research and other interesting sessions, provided satellite programs and an exhibition organized by pharmaceutical companies.

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"Derek Elston, trustee of the ITP Support Association, tells PharmaTimes about his experiences of being diagnosed and living with the rare bleeding disorder.

I was first diagnosed in 1993 with ITP after working in the Far East for 14 months. At the end I was fairly shattered both with the intensity of the work and commuting regularly. After about four weeks, I decided to visit the doctor. He took a sample of blood. This was followed by a frantic telephone call at midday. My platelet count was much lower than normal and he arranged for me to see the haematologist consultant at our local hospital. Normally the platelet count should be between 150 and 450, mine was ten!
After asking many questions and another blood sample, he concluded I had been infected with a virus and it had affected my autoimmune system. This he informed was called idiopathic (now immune) thrombocytopenic purpora or ITP for short, a non-malignant bleeding disorder."

- By Selina McKee for

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