When to consider inherited marrow failure syndromes in adults
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):548-555. doi: 10.1182/hematology.2023000488.ABSTRACTThe inherited bone marrow failure syndromes (IBMFS) are a heterogenous group of disorders caused by germline mutations in related genes and characterized by bone marrow failure (BMF), disease specific organ involvement, and, in most cases, predisposition to malignancy. Their distinction from immune marrow failure can often be challenging, particularly when presentations occur in adulthood or are atypical. A combination of functional (disease specific assays) and genetic testing is optimal in assessing all new BMF ...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Fernanda Gutierrez-Rodrigues Bhavisha A Patel Emma M Groarke Source Type: research

Minimal intensity conditioning strategies for bone marrow failure: is it time for "preventative" transplants?
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):135-140. doi: 10.1182/hematology.2023000470.ABSTRACTHematopoietic cell transplantation (HCT) can cure blood dyscrasias and reduce the risk of hematologic cancers in patients with inherited bone marrow failure syndromes (IBMFS). However, because of its high mortality rate, HCT is generally reserved until patients with IBMFS manifest life-threatening cytopenias or myeloid malignancy, at which point outcomes are poor. Screening tests that accurately predict transformation and enable timely intervention are lacking. These unknowns and risks limit the use of HCT in pati...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Suneet Agarwal Source Type: research

When to consider inherited marrow failure syndromes in adults
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):548-555. doi: 10.1182/hematology.2023000488.ABSTRACTThe inherited bone marrow failure syndromes (IBMFS) are a heterogenous group of disorders caused by germline mutations in related genes and characterized by bone marrow failure (BMF), disease specific organ involvement, and, in most cases, predisposition to malignancy. Their distinction from immune marrow failure can often be challenging, particularly when presentations occur in adulthood or are atypical. A combination of functional (disease specific assays) and genetic testing is optimal in assessing all new BMF ...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Fernanda Gutierrez-Rodrigues Bhavisha A Patel Emma M Groarke Source Type: research

Minimal intensity conditioning strategies for bone marrow failure: is it time for "preventative" transplants?
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):135-140. doi: 10.1182/hematology.2023000470.ABSTRACTHematopoietic cell transplantation (HCT) can cure blood dyscrasias and reduce the risk of hematologic cancers in patients with inherited bone marrow failure syndromes (IBMFS). However, because of its high mortality rate, HCT is generally reserved until patients with IBMFS manifest life-threatening cytopenias or myeloid malignancy, at which point outcomes are poor. Screening tests that accurately predict transformation and enable timely intervention are lacking. These unknowns and risks limit the use of HCT in pati...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Suneet Agarwal Source Type: research

When to consider inherited marrow failure syndromes in adults
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):548-555. doi: 10.1182/hematology.2023000488.ABSTRACTThe inherited bone marrow failure syndromes (IBMFS) are a heterogenous group of disorders caused by germline mutations in related genes and characterized by bone marrow failure (BMF), disease specific organ involvement, and, in most cases, predisposition to malignancy. Their distinction from immune marrow failure can often be challenging, particularly when presentations occur in adulthood or are atypical. A combination of functional (disease specific assays) and genetic testing is optimal in assessing all new BMF ...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Fernanda Gutierrez-Rodrigues Bhavisha A Patel Emma M Groarke Source Type: research

Minimal intensity conditioning strategies for bone marrow failure: is it time for "preventative" transplants?
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):135-140. doi: 10.1182/hematology.2023000470.ABSTRACTHematopoietic cell transplantation (HCT) can cure blood dyscrasias and reduce the risk of hematologic cancers in patients with inherited bone marrow failure syndromes (IBMFS). However, because of its high mortality rate, HCT is generally reserved until patients with IBMFS manifest life-threatening cytopenias or myeloid malignancy, at which point outcomes are poor. Screening tests that accurately predict transformation and enable timely intervention are lacking. These unknowns and risks limit the use of HCT in pati...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Suneet Agarwal Source Type: research

When to consider inherited marrow failure syndromes in adults
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):548-555. doi: 10.1182/hematology.2023000488.ABSTRACTThe inherited bone marrow failure syndromes (IBMFS) are a heterogenous group of disorders caused by germline mutations in related genes and characterized by bone marrow failure (BMF), disease specific organ involvement, and, in most cases, predisposition to malignancy. Their distinction from immune marrow failure can often be challenging, particularly when presentations occur in adulthood or are atypical. A combination of functional (disease specific assays) and genetic testing is optimal in assessing all new BMF ...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Fernanda Gutierrez-Rodrigues Bhavisha A Patel Emma M Groarke Source Type: research

Minimal intensity conditioning strategies for bone marrow failure: is it time for "preventative" transplants?
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):135-140. doi: 10.1182/hematology.2023000470.ABSTRACTHematopoietic cell transplantation (HCT) can cure blood dyscrasias and reduce the risk of hematologic cancers in patients with inherited bone marrow failure syndromes (IBMFS). However, because of its high mortality rate, HCT is generally reserved until patients with IBMFS manifest life-threatening cytopenias or myeloid malignancy, at which point outcomes are poor. Screening tests that accurately predict transformation and enable timely intervention are lacking. These unknowns and risks limit the use of HCT in pati...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Suneet Agarwal Source Type: research

When to consider inherited marrow failure syndromes in adults
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):548-555. doi: 10.1182/hematology.2023000488.ABSTRACTThe inherited bone marrow failure syndromes (IBMFS) are a heterogenous group of disorders caused by germline mutations in related genes and characterized by bone marrow failure (BMF), disease specific organ involvement, and, in most cases, predisposition to malignancy. Their distinction from immune marrow failure can often be challenging, particularly when presentations occur in adulthood or are atypical. A combination of functional (disease specific assays) and genetic testing is optimal in assessing all new BMF ...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Fernanda Gutierrez-Rodrigues Bhavisha A Patel Emma M Groarke Source Type: research

Minimal intensity conditioning strategies for bone marrow failure: is it time for "preventative" transplants?
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):135-140. doi: 10.1182/hematology.2023000470.ABSTRACTHematopoietic cell transplantation (HCT) can cure blood dyscrasias and reduce the risk of hematologic cancers in patients with inherited bone marrow failure syndromes (IBMFS). However, because of its high mortality rate, HCT is generally reserved until patients with IBMFS manifest life-threatening cytopenias or myeloid malignancy, at which point outcomes are poor. Screening tests that accurately predict transformation and enable timely intervention are lacking. These unknowns and risks limit the use of HCT in pati...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Suneet Agarwal Source Type: research

When to consider inherited marrow failure syndromes in adults
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):548-555. doi: 10.1182/hematology.2023000488.ABSTRACTThe inherited bone marrow failure syndromes (IBMFS) are a heterogenous group of disorders caused by germline mutations in related genes and characterized by bone marrow failure (BMF), disease specific organ involvement, and, in most cases, predisposition to malignancy. Their distinction from immune marrow failure can often be challenging, particularly when presentations occur in adulthood or are atypical. A combination of functional (disease specific assays) and genetic testing is optimal in assessing all new BMF ...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Fernanda Gutierrez-Rodrigues Bhavisha A Patel Emma M Groarke Source Type: research

Minimal intensity conditioning strategies for bone marrow failure: is it time for "preventative" transplants?
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):135-140. doi: 10.1182/hematology.2023000470.ABSTRACTHematopoietic cell transplantation (HCT) can cure blood dyscrasias and reduce the risk of hematologic cancers in patients with inherited bone marrow failure syndromes (IBMFS). However, because of its high mortality rate, HCT is generally reserved until patients with IBMFS manifest life-threatening cytopenias or myeloid malignancy, at which point outcomes are poor. Screening tests that accurately predict transformation and enable timely intervention are lacking. These unknowns and risks limit the use of HCT in pati...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Suneet Agarwal Source Type: research

When to consider inherited marrow failure syndromes in adults
Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):548-555. doi: 10.1182/hematology.2023000488.ABSTRACTThe inherited bone marrow failure syndromes (IBMFS) are a heterogenous group of disorders caused by germline mutations in related genes and characterized by bone marrow failure (BMF), disease specific organ involvement, and, in most cases, predisposition to malignancy. Their distinction from immune marrow failure can often be challenging, particularly when presentations occur in adulthood or are atypical. A combination of functional (disease specific assays) and genetic testing is optimal in assessing all new BMF ...
Source: Hematology ASH Education Program - December 9, 2023 Category: Hematology Authors: Fernanda Gutierrez-Rodrigues Bhavisha A Patel Emma M Groarke Source Type: research