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Support Association Inc.

Increasing the understanding of Neutropenia by providing information and support for caregivers, patients and their families.

Corona virus

Corona virus disease 2019 (COVID-19) is a respiratory illness that can spread from person to person. The virus is thought to spread mainly between people who are in close contact with one another (within about 6 feet) through respiratory droplets produced when an infected person coughs or sneezes.

The Corona virus, or COVID-19, has been detected in nearly all countries (all but Antarctica); and over 115,000 has been infected.  Reported illnesses have ranged from mild to severe.  The numbers of those who will test positive for the virus will increase but keep in mind that most of the affected will have mild symptoms.

 

For those with Neutropenia, we think it is best that all follow the CDC guidelines as amplified or tailored locally. These steps include:

 

----  Individuals who are on G-CSF should be sure to be vigilant, maintain dosing and not miss doses.

 ----  For those not on G-CSF, when illness occurs, the clinic or ER providing services should be advised that the patient has neutropenia and get a CBC.

 ----  If neutrophil counts are substantially lower than usual, as occurs with some viral infections, i.e., less than 1000, we recommend G-CSF for at least a few days; work with your physician.

 ----  Regarding travel, as far as we know, there is risk is being in crowds with some with respiratory illness, even in the beginning stages. So, airplanes, large social gatherings, etc. are risky and in general should be avoided. 

 

The standard precautions should be done by everyone, including those with Neutropenia

 

---- Wash your hands often.

 ---- If soap and water are not readily available, use a hand sanitizer that contains at least 60% alcohol. Cover all surfaces of your hands and rub them together until they feel dry.

 ---- Avoid touching your eyes, nose, and mouth with unwashed hands.

 ---- Avoid close contact with people who are sick

 ---- Put distance between yourself and other people if COVID-19 is spreading in your community.

 ---- Cover your mouth and nose with a tissue when you cough or sneeze or use the inside of your elbow.

 ---- Throw used tissues in the trash.

 ---- Wear a face mask if you are sick: You should wear a face mask when you are around other people (e.g., sharing a room or vehicle) and before you enter a healthcare provider’s office. If you are not able to wear a face mask (for example, because it causes trouble breathing), then you should do your best to cover your coughs and sneezes, and people who are caring for you should wear a face mask if they enter your room.

 ---- If you are NOT sick: You do not need to wear a face mask unless you are caring for someone who is sick (and they are not able to wear a face mask). Face masks may be in short supply and they should be saved for caregivers.

 ---- Clean and disinfect frequently touched surfaces daily. This includes tables, doorknobs, light switches, counter tops, handles, desks, phones, keyboards, toilets, faucets, and sinks.

 ---- If surfaces are dirty, clean them: Use detergent or soap and water prior to disinfection.

 ---- If you do get sick, seek treatment.

 

While the immediate risk of COVID-19 is  still low at this time, there is still a much more immediate threat of influenza. It's currently flu and respiratory disease season and CDC recommends getting a flu vaccine and taking everyday preventive actions to help stop the spread of germs. If you have not received your yearly flu shot, it’s not too late to get one.

 

This is an emerging, rapidly evolving situation and CDC will continue to provide updated information as it becomes available. For additional and updated information, visit CDC’s Coronavirus 2019 website pages.

 

Stay well.

 

*This information was written and organized by the National Neutropenia Network and the Neutropenia experts with the SCNIR for those who subscribe to the National Neutropenia Network's newsletter. This information is not intended to serve as medical advice. Please see your physician or seek medical treatment is you are ill. 

4whim Clinical Trial Info

Clinical Trial Information

 

X4 Pharmaceutical’s (Cambridge, MA) Phase 3 WHIM trial is open now for enrollment, entitled “A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Trial of mavorixafor (X4P-001) in Patients with WHIM Syndrome with Open-Label Extension

WHIM (Warts, Hypogammaglobulinemia, Infections and Myelokathexis) is a rare autosomal dominant primary immunodeficiency disease caused by gain-of-function mutations in the chemokine receptor CXCR4.  CXCR4 mutations induce increased leukocyte homing and retention in the bone marrow resulting in severe chronic neutropenia and lymphopenia.

X4 Pharmaceuticals is planning to sponsor a 52-week global Phase 3 study to evaluate the safety and efficacy of mavorixafor, a once-daily oral CXCR4 antagonist, in genetically confirmed WHIM patients who are aged 12 years or older.  Mavorixafor is a first-in-class, oral, small molecule antagonist of the CXCR4 chemokine receptor that has demonstrated a favorable safety profile and dose-dependent increases in ANC and ALC in a Phase 2 study. Trial subjects will participate in a one-year trial with an opportunity to continue in the subsequent open label extension.  Travel support to study sites will be provided.

To support confirming the diagnosis of WHIM, X4 Pharmaceuticals will offer genetic testing at no charge to patients meeting eligibility criteria suggestive for the disease.  For information and support for genetic confirmation of your patients with suspected WHIM, please contact This email address is being protected from spambots. You need JavaScript enabled to view it.

 

Phase 1b Clinical Trial for the Treatment of Severe Congenital and Chronic Idiopathic Neutropenia

X4 Pharmaceuticals, Inc (Cambridge, MA), a clinical-stage biopharmaceutical company focused on the development of novel therapeutics for the treatment of rare diseases, announced the initiation of a Phase 1b open-label, multicenter clinical trial titled: A Study of Mavorixafor in Participants with Severe Congenital. Neutropenia and Chronic Neutropenia Disorders. Visit their web for more information: www.clinicaltrials.gov/ct2/show/NCT04154488.

The Phase 1b trial is a 14-day, proof-of-concept trial designed to assess the safety and tolerability of daily, oral mavorixafor – an investigational therapy – in up to 45 participants with Severe Congenital Neutropenia (SCN) disorders or Chronic Idiopathic Neutropenia (CIN). In addition, the trial will evaluate the neutrophil response, of mavorixafor, as an independent agent or in combination with granulocyte-colony stimulating factor (G-CSF). SCN are rare disorders; genetic testing can help to confirm the diagnosis and enhance patient access to appropriate care. Through its collaborative PATH4WARD program, X4 provides genetic testing at no cost to patients with severe neutropenia. This initiative provides greater access to faster and earlier diagnosis for individuals who may carry a genetic mutation known to be associated with SCN.

For inquiries about the clinical trial and/or PATH4WARD genetic testing program, please contact X4 pharmaceuticals at This email address is being protected from spambots. You need JavaScript enabled to view it..
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What We Do

What We Do

The Neutropenia Support Assoc. is a passionate advocate for patients with chronic illness with regard to access issues, patient's needs, patient's protection, and patient's rights. The association has many articles on neutropenia and it's treatment. We maintain an information library to answer questions from patients, their families, and the medical community. We also raise seed money for research.

Some of the groups we are voluntarily working with right now are the Technical Committee on Blood and Blood Components CSA. Other areas of interest are drug strategies and guidelines, drug approval, long term drug safety surveillance, bone marrow transplantation and genetics studies.

About Neutropenia

About Neutropenia

Neutropenia is a blood disorder you don't often hear about, but it can affect anyone. Some people are born with it. It can happen after a viral infection, or can be drug induced. Some offending agents include some antibiotics, antithyroid agents, tranquilizers, anti-inflammatory agents, antirheumatics, sedatives etc. Exposure to certain poisons can also cause Neutropenia.

Neutropenia affects as many as one in three patients receiving chemotherapy for cancer. It is also associated with many other diseases like Lupus (SLE), Malaria, Hepatitis viruses, Barth Syndrome, Rheumatoid Arthritis, Sjogren's Syndrome, Shwachman-Diamond Syndrome, Aplastic Anemia and Myelodysplastic Syndromes, Parvovirus, Felty's Syndrome, to name just a few.

Neutropenia can be a serious problem requiring prompt attention. Without proper medical care, patients may find it hard to lead normal lives. Severe cases can be life threatening.

Other rare diseases like Shwachman-Diamond Syndrome, Fanconi Anemia, and Kostmann's Syndrome (a congenital neutropenia) can evolve to leukemia and require a bone marrow transplant. The development of treatments and the study of other diseases like AML leukemia, have led to involvement with many organizations which share similar issues.

Who We Are

The Neutropenia Support Association Inc. is a volunteer driven registered charity, that was formed in 1989 to increase awareness and understanding of Neutropenia by providing assistance to Neutropenia patients and their families, and raise money for research and education.