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Article ; Online: Congenital and acquired bleeding disorders in pregnancy.

Gernsheimer, Terry B

Hematology. American Society of Hematology. Education Program

2016 Volume 2016, Issue 1, Page(s) 232–235

Abstract: Obstetrical hematology represents challenges not only for the patient, but also for her progeny. In particular, bleeding disorders, both congenital and acquired, not only present problems both for delivery and in the immediate postpartum period, but also ...

Abstract	Obstetrical hematology represents challenges not only for the patient, but also for her progeny. In particular, bleeding disorders, both congenital and acquired, not only present problems both for delivery and in the immediate postpartum period, but also may have significant implications for the fetus and neonate. Women with congenital bleeding disorders or who are carriers of X-linked or autosomal disorders should be counseled prior to conception so that pregnancy can be safely undertaken with careful preparation. A treatment plan should be set up by a specialized care team that includes the hematologist, obstetrician, and anesthesiologist; the patient should be followed closely during pregnancy, through delivery, and in the immediate postpartum period. Acquired disorders of hemostasis that occur with pregnancy may present particular diagnostic difficulties and require rapid diagnosis and management.
MeSH term(s)	Blood Coagulation Disorders, Inherited/diagnosis ; Blood Coagulation Disorders, Inherited/therapy ; Delivery, Obstetric ; Female ; Humans ; Postpartum Period ; Pregnancy ; Pregnancy Complications, Hematologic/diagnosis ; Pregnancy Complications, Hematologic/therapy
Language	English
Publishing date	2016-11-30
Publishing country	United States
Document type	Journal Article ; Review
ISSN	1520-4383
ISSN (online)	1520-4383
DOI	10.1182/asheducation-2016.1.232
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Article ; Online: Glucocorticoids promote response to thrombopoietin-receptor agonists in refractory ITP: a case series.

Poston, Jacqueline N / Gernsheimer, Terry B

International journal of hematology

2019 Volume 110, Issue 2, Page(s) 255–259

Abstract: A proportion of patients with immune thrombocytopenic purpura are refractory to multiple therapies including thrombopoietin-receptor agonists (TPO-RA). We report 10 patients who did not respond to a TPO-RA until the addition of a glucocorticoid. These ... ...

Abstract	A proportion of patients with immune thrombocytopenic purpura are refractory to multiple therapies including thrombopoietin-receptor agonists (TPO-RA). We report 10 patients who did not respond to a TPO-RA until the addition of a glucocorticoid. These patients were previously treated with a median of 6 therapies. One patient elected to discontinue both medications despite persistent thrombocytopenia. The remaining 9 patients continued on the combination of prednisone (doses 5 mg every other day to 10 mg daily) and a TPO-RA. Combination therapy with low dose glucocorticoid and a TPO-RA may be an option for patients unresponsive to a TPO-RA alone.
MeSH term(s)	Adolescent ; Adult ; Aged ; Benzoates/administration & dosage ; Benzoates/adverse effects ; Benzoates/pharmacology ; Benzoates/therapeutic use ; Child, Preschool ; Combined Modality Therapy ; Drug Synergism ; Drug Therapy, Combination ; Female ; Humans ; Hydrazines/administration & dosage ; Hydrazines/adverse effects ; Hydrazines/pharmacology ; Hydrazines/therapeutic use ; Male ; Middle Aged ; Platelet Count ; Prednisone/administration & dosage ; Prednisone/adverse effects ; Prednisone/pharmacology ; Prednisone/therapeutic use ; Purpura, Thrombocytopenic, Idiopathic/drug therapy ; Purpura, Thrombocytopenic, Idiopathic/surgery ; Pyrazoles/administration & dosage ; Pyrazoles/adverse effects ; Pyrazoles/pharmacology ; Pyrazoles/therapeutic use ; Receptors, Fc/administration & dosage ; Receptors, Fc/therapeutic use ; Receptors, Thrombopoietin/antagonists & inhibitors ; Recombinant Fusion Proteins/administration & dosage ; Recombinant Fusion Proteins/adverse effects ; Recombinant Fusion Proteins/pharmacology ; Recombinant Fusion Proteins/therapeutic use ; Splenectomy ; Thrombopoietin/administration & dosage ; Thrombopoietin/adverse effects ; Thrombopoietin/pharmacology ; Thrombopoietin/therapeutic use
Chemical Substances	Benzoates ; Hydrazines ; Pyrazoles ; Receptors, Fc ; Receptors, Thrombopoietin ; Recombinant Fusion Proteins ; MPL protein, human (143641-95-6) ; Thrombopoietin (9014-42-0) ; romiplostim (GN5XU2DXKV) ; eltrombopag (S56D65XJ9G) ; Prednisone (VB0R961HZT)
Language	English
Publishing date	2019-04-10
Publishing country	Japan
Document type	Journal Article
ZDB-ID	1076875-0
ISSN	1865-3774 ; 0917-1258 ; 0925-5710
ISSN (online)	1865-3774
ISSN	0917-1258 ; 0925-5710
DOI	10.1007/s12185-019-02638-6
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Article ; Online: Additional efficacy analysis of avatrombopag phase III data for the treatment of adults with immune thrombocytopenia.

Jain, Shivi / Gernsheimer, Terry / Kolodny, Scott / Bernheisel, Chelsea / Vredenburg, Michael / Panch, Sandhya R

Platelets

2023 Volume 34, Issue 1, Page(s) 2195016

Abstract: Avatrombopag is an oral thrombopoietin receptor agonist (TPO-RA) that was approved in the US in 2019 for treatment of chronic immune thrombocytopenia (ITP). This post hoc analysis of the pivotal phase III study (NCT01438840) of avatrombopag in adult ... ...

Abstract	Avatrombopag is an oral thrombopoietin receptor agonist (TPO-RA) that was approved in the US in 2019 for treatment of chronic immune thrombocytopenia (ITP). This post hoc analysis of the pivotal phase III study (NCT01438840) of avatrombopag in adult patients with ITP evaluated platelet count response to avatrombopag during the core study in different subgroups, and durability of response data in patients who responded to avatrombopag treatment both during the core phase (total population) and during the core and extension phase (total population and by subgroup). Loss of response (LOR [platelet count <30 × 10
MeSH term(s)	Humans ; Adult ; Purpura, Thrombocytopenic, Idiopathic/drug therapy ; Purpura, Thrombocytopenic, Idiopathic/chemically induced ; Receptors, Thrombopoietin/agonists ; Thrombocytopenia/drug therapy ; Thrombocytopenia/chemically induced ; Platelet Count ; Thrombopoietin/adverse effects ; Recombinant Fusion Proteins
Chemical Substances	avatrombopag (3H8GSZ4SQL) ; Receptors, Thrombopoietin ; Thrombopoietin (9014-42-0) ; Recombinant Fusion Proteins
Language	English
Publishing date	2023-04-03
Publishing country	England
Document type	Journal Article
ZDB-ID	1034283-7
ISSN	1369-1635 ; 0953-7104
ISSN (online)	1369-1635
ISSN	0953-7104
DOI	10.1080/09537104.2023.2195016
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Article ; Online: How I treat primary ITP in adult patients who are unresponsive to or dependent on corticosteroid treatment.

Ghanima, Waleed / Gernsheimer, Terry / Kuter, David J

Blood

2021 Volume 137, Issue 20, Page(s) 2736–2744

Abstract: Approximately 80% of adult patients with immune thrombocytopenia (ITP) have treatment failure with corticosteroids or become dependent on them and require second-line therapy. Several new and effective therapies have been introduced during the past ... ...

Abstract	Approximately 80% of adult patients with immune thrombocytopenia (ITP) have treatment failure with corticosteroids or become dependent on them and require second-line therapy. Several new and effective therapies have been introduced during the past decade and our understanding of disease burden and its effect on quality of life has expanded. It is now recommended that splenectomy, the standard second-line therapy for decades, be delayed for at least 12 to 24 months, allowing for more patients to achieve remission on medical therapies before considering surgery. It is highly recommended that medical therapies be used that have abundant clinical trial evidence, such as the thrombopoietin receptor agonists (TPO-RAs) rituximab and fostamatinib. Unfortunately, there are no reliable biomarkers that help in treatment selection. These therapeutic medical options have variable efficacy, safety profiles, mechanisms of action, and modes of administration. This enables and mandates an individualized approach to treatment, where patient involvement, preferences and values have become central to the process of choosing the appropriate therapy. Both TPO-RAs and fostamatinib are maintenance therapies, whereas rituximab is given for a limited number of doses. Although the response is usually maintained while receiving a TPO-RA or fostamatinib therapy, half of rituximab responders will no longer respond 1 to 2 years after administration and require retreatment or other therapy.
MeSH term(s)	Adrenal Cortex Hormones/therapeutic use ; Adult ; Aged ; Aminopyridines/administration & dosage ; Aminopyridines/therapeutic use ; Combined Modality Therapy ; Disease Management ; Drug Substitution ; Drug Tolerance ; Elective Surgical Procedures ; Female ; Hemorrhage/etiology ; Hemorrhage/prevention & control ; Humans ; Immunosuppressive Agents/therapeutic use ; Maintenance Chemotherapy ; Male ; Middle Aged ; Morpholines/administration & dosage ; Morpholines/therapeutic use ; Preoperative Care ; Purpura, Thrombocytopenic, Idiopathic/complications ; Purpura, Thrombocytopenic, Idiopathic/drug therapy ; Pyrimidines/administration & dosage ; Pyrimidines/therapeutic use ; Randomized Controlled Trials as Topic ; Receptors, Fc/therapeutic use ; Receptors, Thrombopoietin/agonists ; Recombinant Fusion Proteins/therapeutic use ; Remission Induction ; Rituximab/administration & dosage ; Rituximab/therapeutic use ; Splenectomy ; Thrombopoietin/therapeutic use ; Young Adult
Chemical Substances	Adrenal Cortex Hormones ; Aminopyridines ; Immunosuppressive Agents ; Morpholines ; Pyrimidines ; Receptors, Fc ; Receptors, Thrombopoietin ; Recombinant Fusion Proteins ; MPL protein, human (143641-95-6) ; Rituximab (4F4X42SYQ6) ; Thrombopoietin (9014-42-0) ; romiplostim (GN5XU2DXKV) ; fostamatinib (SQ8A3S5101)
Language	English
Publishing date	2021-04-07
Publishing country	United States
Document type	Case Reports ; Journal Article
ZDB-ID	80069-7
ISSN	1528-0020 ; 0006-4971
ISSN (online)	1528-0020
ISSN	0006-4971
DOI	10.1182/blood.2021010968
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Article ; Online: Accidental massive platelet transfusion.

Tsang, Hamilton C / Gernsheimer, Terry B / Abkowitz, Janis L / Hess, John R

Transfusion

2021 Volume 61, Issue 5, Page(s) 1359–1360

MeSH term(s)	Blood Platelets/cytology ; Brain Neoplasms/diagnosis ; Brain Neoplasms/surgery ; Humans ; Male ; Middle Aged ; Platelet Count ; Platelet Transfusion/methods ; Preoperative Care ; Splenomegaly/diagnosis
Language	English
Publishing date	2021-02-11
Publishing country	United States
Document type	Case Reports ; Journal Article
ZDB-ID	208417-x
ISSN	1537-2995 ; 0041-1132
ISSN (online)	1537-2995
ISSN	0041-1132
DOI	10.1111/trf.16314
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Zs.A 284: Show issues

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Article ; Online: Durability of platelet response after switching to avatrombopag from eltrombopag or romiplostim in immune thrombocytopenia.

Al-Samkari, Hanny / Jiang, Debbie / Gernsheimer, Terry / Liebman, Howard / Lee, Susie / Bernheisel, Chelsea / Wojdyla, Matthew / Vredenburg, Michael / Cuker, Adam

Research and practice in thrombosis and haemostasis

2023 Volume 7, Issue 3, Page(s) 100134

Language	English
Publishing date	2023-03-29
Publishing country	United States
Document type	Journal Article
ISSN	2475-0379
ISSN (online)	2475-0379
DOI	10.1016/j.rpth.2023.100134
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Article ; Online: Evaluating the prevalence of inborn errors of immunity in adults with chronic immune thrombocytopenia or Evans syndrome.

Jiang, Debbie / Rosenlind, Kira / Baxter, Sarah / Gernsheimer, Terry / Gulsuner, Suleyman / Allenspach, Eric J / Keel, Siobán B

Blood advances

2023 Volume 7, Issue 23, Page(s) 7202–7208

Abstract: Inborn errors of immunity (IEIs) are monogenic disorders that predispose patients to immune dysregulation, autoimmunity, and infection. Autoimmune cytopenias, such as immune thrombocytopenia (ITP) and Evans syndrome (a combination of ITP and autoimmune ... ...

Abstract	Inborn errors of immunity (IEIs) are monogenic disorders that predispose patients to immune dysregulation, autoimmunity, and infection. Autoimmune cytopenias, such as immune thrombocytopenia (ITP) and Evans syndrome (a combination of ITP and autoimmune hemolytic anemia), are increasingly recognized phenotypes of IEI. Although recent findings suggest that IEIs may commonly underlie pediatric ITP and Evans syndrome, its prevalence in adult patients with these disorders remains undefined. This study sought to estimate the prevalence of underlying IEIs among adults with persistent or chronic ITP or Evans syndrome using a next-generation sequencing panel encompassing >370 genes implicated in IEIs. Forty-four subjects were enrolled from an outpatient adult hematology clinic at a tertiary referral center in the United States, with a median age of 49 years (range, 20-83). Fourteen subjects (31.8%) had secondary ITP, including 8 (18.2%) with Evans syndrome. No cases of IEI were identified despite a high representation of subjects with a personal history of autoimmunity (45.5%) and early onset of disease (median age at diagnosis of 40 years [range, 2-77]), including 20.5% who were initially diagnosed as children. Eight subjects (18.2%) were found to be carriers of pathogenic IEI variants, which, in their heterozygous state, are not disease-causing. One case of TUBB1-related congenital thrombocytopenia was identified. Although systematic screening for IEI has been proposed for pediatric patients with Evans syndrome, findings from this real-world study suggest that inclusion of genetic testing for IEI in the routine work-up of adults with ITP and Evans syndrome has a low diagnostic yield.
MeSH term(s)	Humans ; Adult ; Child ; Young Adult ; Middle Aged ; Aged ; Aged, 80 and over ; Child, Preschool ; Adolescent ; Anemia, Hemolytic, Autoimmune/epidemiology ; Anemia, Hemolytic, Autoimmune/genetics ; Anemia, Hemolytic, Autoimmune/complications ; Purpura, Thrombocytopenic, Idiopathic/epidemiology ; Purpura, Thrombocytopenic, Idiopathic/genetics ; Purpura, Thrombocytopenic, Idiopathic/complications ; Autoimmunity ; Prevalence ; Thrombocytopenia/epidemiology ; Thrombocytopenia/genetics ; Thrombocytopenia/complications
Language	English
Publishing date	2023-10-04
Publishing country	United States
Document type	Journal Article ; Research Support, N.I.H., Extramural
ZDB-ID	2915908-8
ISSN	2473-9537 ; 2473-9529
ISSN (online)	2473-9537
ISSN	2473-9529
DOI	10.1182/bloodadvances.2023011042
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Article ; Online: Clinical updates in adult immune thrombocytopenia.

Lambert, Michele P / Gernsheimer, Terry B

Blood

2017 Volume 129, Issue 21, Page(s) 2829–2835

Abstract: Immune thrombocytopenia (ITP) occurs in 2 to 4/100 000 adults and results in variable bleeding symptoms and thrombocytopenia. In the last decade, changes in our understanding of the pathophysiology of the disorder have led to the publication of new ... ...

Abstract	Immune thrombocytopenia (ITP) occurs in 2 to 4/100 000 adults and results in variable bleeding symptoms and thrombocytopenia. In the last decade, changes in our understanding of the pathophysiology of the disorder have led to the publication of new guidelines for the diagnosis and management of ITP and standards for terminology. Current evidence supports alternatives to splenectomy for second-line management of patients with persistently low platelet counts and bleeding. Long-term follow-up data suggest both efficacy and safety, in particular, for the thrombopoietin receptor agonists and the occurrence of late remissions. Follow-up of patients who have undergone splenectomy for ITP reveals significant potential risks that should be discussed with patients and may influence clinician and patient choice of second-line therapy. Novel therapeutics are in development to address ongoing treatment gaps.
MeSH term(s)	Adult ; Female ; Hemorrhage/blood ; Hemorrhage/diagnosis ; Hemorrhage/physiopathology ; Hemorrhage/therapy ; Humans ; Male ; Platelet Count ; Practice Guidelines as Topic ; Purpura, Thrombocytopenic, Idiopathic/blood ; Purpura, Thrombocytopenic, Idiopathic/diagnosis ; Purpura, Thrombocytopenic, Idiopathic/physiopathology ; Purpura, Thrombocytopenic, Idiopathic/therapy ; Receptors, Thrombopoietin/agonists ; Receptors, Thrombopoietin/metabolism ; Splenectomy
Chemical Substances	Receptors, Thrombopoietin ; MPL protein, human (143641-95-6)
Language	English
Publishing date	2017-04-17
Publishing country	United States
Document type	Journal Article ; Review ; Research Support, N.I.H., Extramural
ZDB-ID	80069-7
ISSN	1528-0020 ; 0006-4971
ISSN (online)	1528-0020
ISSN	0006-4971
DOI	10.1182/blood-2017-03-754119
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Article ; Online: Thrombocytopenia in pregnancy: is this immune thrombocytopenia or...?

Gernsheimer, Terry B

Hematology. American Society of Hematology. Education Program

2012 Volume 2012, Page(s) 198–202

Abstract: Thrombocytopenia is a common finding in pregnancy. Establishing the diagnosis of immune thrombocytopenia (ITP) in a pregnant patient is similar to doing so in a nonpregnant patient, except that the evaluation must specifically rule out other disorders of ...

Abstract	Thrombocytopenia is a common finding in pregnancy. Establishing the diagnosis of immune thrombocytopenia (ITP) in a pregnant patient is similar to doing so in a nonpregnant patient, except that the evaluation must specifically rule out other disorders of pregnancy associated with low platelet counts that present different risks to the mother and fetus and may require alternate distinct therapy. Many of the same treatment modalities are used to manage the pregnant patient with ITP, but others have not been determined to be safe for the fetus, are limited to a particular gestational period, or side effects may be more problematic during pregnancy. The therapeutic objective differs from that in chronic ITP in the adult because many pregnant patients recover or improve spontaneously after delivery and therefore maintenance of a safe platelet count, rather than prolonged remission, is the goal. Thrombocytopenia may the limit choices of anesthesia, but does not guide mode of delivery, and the fetus is rarely severely affected at birth. Patients should be advised that a history of ITP or ITP in a previous pregnancy is not a contraindication to future pregnancies and that, with proper management and monitoring, positive outcomes can be expected in the majority of patients.
MeSH term(s)	Adult ; Delivery, Obstetric ; Diagnosis, Differential ; Female ; Humans ; Immune System ; Infant, Newborn ; Obstetrics/methods ; Platelet Count ; Pregnancy ; Pregnancy Complications, Hematologic/diagnosis ; Risk ; Thrombocytopenia/diagnosis ; Thrombocytopenia/immunology ; Treatment Outcome
Language	English
Publishing date	2012
Publishing country	United States
Document type	Journal Article ; Research Support, Non-U.S. Gov't ; Review
ISSN	1520-4383
ISSN (online)	1520-4383
DOI	10.1182/asheducation-2012.1.198
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Article ; Online: Chronic idiopathic thrombocytopenic purpura: mechanisms of pathogenesis.

Gernsheimer, Terry

The oncologist

2009 Volume 14, Issue 1, Page(s) 12–21

Abstract: The mechanism of idiopathic (autoimmune) thrombocytopenic purpura (ITP) has historically been attributed to platelet autoantibody production and the resultant platelet destruction. More recent evidence suggests a multifactorial pathogenesis. A complex ... ...

Abstract	The mechanism of idiopathic (autoimmune) thrombocytopenic purpura (ITP) has historically been attributed to platelet autoantibody production and the resultant platelet destruction. More recent evidence suggests a multifactorial pathogenesis. A complex picture of the immune processes involved in autoimmunity has emerged over the last decade with the identification and characterization of immunoregulatory elements (receptors, cytokines, and other signaling molecules) and cell trafficking patterns. An understanding of the interplay of cellular and humoral immune responses in the breakdown of self-tolerance has brought to light unrecognized mechanisms of the autoimmune destruction of platelets in ITP and potential targets for future therapeutic advances. The failure of the bone marrow to maximally increase platelet production also appears to play an important role in the thrombocytopenia of ITP. Treatment strategies targeting the thrombopoietin receptor to increase platelet production are a promising new approach to the management of ITP.
MeSH term(s)	Blood Platelets/immunology ; Humans ; Purpura, Thrombocytopenic, Idiopathic/blood ; Purpura, Thrombocytopenic, Idiopathic/immunology
Language	English
Publishing date	2009-01
Publishing country	United States
Document type	Journal Article ; Research Support, Non-U.S. Gov't ; Review
ZDB-ID	1409038-7
ISSN	1549-490X ; 1083-7159
ISSN (online)	1549-490X
ISSN	1083-7159
DOI	10.1634/theoncologist.2008-0132
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