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Hematology Center of Excellence

Supporting patients living with a rare hematology condition

Orsini’s Hematology Center of Excellence launched in 2016 with our first program for paroxysmal nocturnal hemoglobinuria. Today we support multiple hematology programs across several rare conditions. Our dedicated Therapy Care Teams work with the physician’s office and insurance on a patient’s behalf so that our patients and their family have more time to focus on their treatment and quality of life. We are here to listen, provide counseling on treatment, including side-effect management, and assist with sourcing third-party financial assistance.

Explore Our Hematology Therapies

Benefits

Improving the Patient Experience, Every Time

Dedicated Care Team

Receive personalized support from a dedicated Therapy Care Team, including a primary pharmacy contact, clinical education, side-effects management, and scheduled refill calls.

Home Health Nursing

Access nationwide nursing support for in-home treatment or self-administration training. Our nurses provide clinical support to help patients achieve their treatment goals.

Insurance Coverage

Secure treatment coverage with assistance navigating payor and plan requirements. Our team proactively works with prescribers to obtain prior authorization and support appeals.

Free Care Overnight Shipping

Ensure medication and essential medical supplies arrive when and where patients need them through coordination across prescribers, patients, and care teams to arrange free overnight shipping.

Financial Assistance

Minimize financial exposure through financial assistance programs. We successfully identify and assist in enrolling patients into manufacturer co-pay and foundation support programs.

24/7/365 Access to a Pharmacist

Gain continuous access to a pharmacist, 24/7/365, ensuring ongoing care and clinical support for patients and prescribers to achieve better outcomes.

Support

Hematology Conditions Supported

Explore the rare hematology conditions and therapies Orsini supports below.

About Atypical Hemolytic Uremic Syndrome (aHUS)

Atypical Hemolytic-uremic Syndrome (aHUS) is a disease characterized by low red blood cell count, low platelet count and inability to process and excrete waste products from the blood. Symptoms result from tiny blood clots that form in small blood vessels in the body which impede proper blood flow to various organs of the body, especially the kidneys. As a result, people with atypical hemolytic-uremic syndrome experience kidney damage and acute kidney failure that lead to end-stage renal disease in about half of all cases.

aHUS Therapies Supported

About Congenital Thrombotic Thrombocytopenic Purpura (c-TTP)

Congenital thrombotic thrombocytopenic purpura (c-TTP) is a rare and inherited blood disorder characterized by deficient activity of the enzyme ADAMTS13. This leads to the formation of blood clots in small blood vessels throughout the body, causing thrombocytopenia (low platelet count) and other complications. The lack of properly functioning ADAMTS13 impairs the breakdown of a specific blood clotting factor, resulting in the formation of microclots. c-TTP’s symptoms include purpura, neurological abnormalities, and organ damage. This lifelong condition poses challenges in maintaining blood clotting balance.

c-TTP Disease Therapies Supported

Full Name Adzynma (ADAMTS13, recombinant-krhn)
Drug Adzynma
Manufacturer Takeda Pharmaceuticals U.S.A., Inc.
Route of Administration Intravenous
Site of Care Home or Healthcare Facility
Approved Indication Prophylactic or on demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP)
Disease Congenital thrombotic thrombocytopenic purpura (c-TTP)
Therapeutic Area Hematology
Enrollment Form Link Enrollment Form
Phone Number 800-720-0789
Fax Number 877-251-0709
Product Website adzynma.com

About Hemophilia A

Hemophilia A, also called factor VIII deficiency or classic Hemophilia, is an X-linked genetic disorder caused by missing or defective factor VIII, a clotting protein. Approximately 1 in 10,000 people have Hemophilia A. Individuals with the most severe form of hemophilia A make up approximately 50% of the Hemophilia A population. 

Hemophilia A Disease Therapies Supported

Full Name Roctavian™ (valoctocogene roxaparvovec-rvox)
Drug Roctavian
Manufacturer BioMarin Pharmaceutical Inc.
Route of Administration Intravenous
Site of Care Healthcare Facility
Approved Indication The treatment of adults with severe hemophilia A (congenital factor VIII deficiency with factor VIII activity < 1 IU/dL) without pre-existing antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test
Disease Hemophilia A
Therapeutic Area Hematology; Cell & Gene Therapy
Enrollment Form Link Roctavian Enrollment Form
Phone Number 800-358-0013
Fax Number 877-631-9228
Product Website roctavian.com

About Hemophilia B

Hemophilia B, also known as Christmas disease or factor IX deficiency, is a rare, degenerative disease that occurs in approximately 1 in 25,000 male births. People with this condition are particularly vulnerable to bleeds in their joints, muscles, and internal organs, leading to pain, swelling, and joint damage. Hemophilia B can range from mild (factor IX levels between 5 and 40% of normal) to moderate (factor IX levels from 1 to 5% of normal) to severe (factor IX levels less than 1% of normal).

Hemophilia B Therapies Supported

Full Name Hemgenix® (etranacogene dezaparvovec-drlb)
Drug Hemgenix
Manufacturer CSL Behring
Route of Administration Intravenous
Site of Care Healthcare Facility
Approved Indication The treatment of adults with Hemophilia B (congenital Factor IX deficiency) who currently use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes
Disease Hemophilia B
Therapeutic Area Hematology; Cell & Gene Therapy
Enrollment Form Link Enrollment Form
Phone Number 800-975-8693
Fax Number 877-740-7535
Product Website hemgenix.com/hcp

About Paroxysmal Nocturnal Hemoglobinuria (PNH)

CHAPLE disease (also known as CD55 deficiency with hyperactivation of complement, angiopathic thrombosis, and protein losing enteropathy, or CD55-deficient protein-losing enteropathy) is an ultra-rare hereditary condition characterized by an inability to regulate complement activity because of mutations in the CD55 gene. In most patients, this leads to potentially life-threatening symptoms beginning in infancy, including abdominal pain, bloody diarrhea, vomiting, malnutrition, slow growth, and edema. There are fewer than 100 patients worldwide who are known to have CHAPLE disease.

PNH Therapies Supported

About Sickle Cell Disease (SCD)

Sickle cell disease is an inherited blood disorder causing red blood cells to become “sickled,” leading to severe pain, organ damage, and shorter life expectancy. Hallmarked by painful blood vessel blockages (vaso-occlusive crises, or VOCs), SCD can also cause acute chest syndrome, stroke, jaundice, and heart failure symptoms.

SCD Therapies Supported

Full Name CASGEVY™ (exagamglogene autotemcel)
Drug Casgevy
Manufacturer Vertex Pharmaceuticals
Route of Administration Intravenous
Site of Care Healthcare Facility
Approved Indication Treatment of patients aged 12 years and older with:
  • Sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs)
  • Transfusion-dependent β-thalassemia (TDT)
Disease Sickle cell disease (SCD) and Transfusion-dependent β-thalassemia (TDT)
Therapeutic Area Hematology; Cell & Gene Therapy
Phone Number 1-833-837-8395

About Transfusion-Dependent Beta Thalassemia (TDT)

Transfusion-dependent beta thalassemia is an inherited blood disorder causing anemia, resulting in fatigue, shortness of breath, and, in infants, growth issues and jaundice. Complications can include an enlarged spleen, liver, or heart; misshapen bones; and delayed puberty. Many patients need regular blood transfusions, requiring frequent hospital visits and often causing iron buildup.

TDT Therapies Supported

Full Name CASGEVY™ (exagamglogene autotemcel)
Drug Casgevy
Manufacturer Vertex Pharmaceuticals
Route of Administration Intravenous
Site of Care Healthcare Facility
Approved Indication Treatment of patients aged 12 years and older with:
  • Sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs)
  • Transfusion-dependent β-thalassemia (TDT)
Disease Sickle cell disease (SCD) and Transfusion-dependent β-thalassemia (TDT)
Therapeutic Area Hematology; Cell & Gene Therapy
Phone Number 1-833-837-8395

About Venous Thromboembolism (VTE)

Venous Thromboembolism (VTE) is a blood clot that starts in a vein. It is an underdiagnosed and serious yet preventable medical condition. While VTE is rare in children, VTE in pediatrics is becoming a recognized cause of significant morbidity and mortality in children. Most children diagnosed with VTE have a serious underlying illness such as cancer, chronic total parenteral nutrition dependency, or congenital heart disease.

VTE Therapies Supported

Full Name Pradaxa® (dabigatran etexilate) oral pellets - pediatric
Drug Pradaxa Pediatric
Manufacturer Boehringer Ingelheim
Route of Administration Oral
Site of Care Home
Approved Indication To reduce the risk of stroke and systemic embolism in adult patients with non-valvular atrial fibrillation, for the treatment of deep venous thrombosis (DVT) and pulmonary embolism (PE) in adult patients who have been treated with a parenteral anticoagulant for 5-10 days, to reduce the risk of recurrence of DVT and PE in adult patients who have been previously treated, for the prophylaxis of DVT and PE in adult patients who have undergone hip replacement surgery, for the treatment of venous thromboembolic events (VTE) in pediatric patients 8 to less than 18 years of age who have been treated with a parenteral anticoagulant for at least 5 days, or to reduce the risk of recurrence of VTE in pediatric patients 8 to less than 18 years of age who have been previously treated
Disease Venous Thromboembolism (VTE)
Therapeutic Area Hematology
Enrollment Form Link Pradaxa Enrollment Form
Phone Number 800-593-0310
Fax Number 877-709-9184
Product Website https://patientpatient.boehringer-ingelheim.com/us/pradaxa/