2019 sickle cell disease guidelines by the American Society of Hematology: methodology, challenges, and innovations

Murad, M. Hassan; Liem, Robert I.; Lang, Eddy; Akl, Elie A.; Meerpohl, Joerg J; DeBaun, Michael R.; Tisdale, John F.; Brandow, Amanda M.; Lanzkron, Sophie; Chou, Stella T.; Webb, Starr; Mustafa, Reem A.

doi:10.1182/bloodadvances.2019000931

“…The results observed in this study are similar to those obtained in the small open‐labeled studies of sildenafil use in SCD‐PH 12,16 . Those studies demonstrated potential utility of sildenafil and served to lay a framework for the design of the multicenter randomized, placebo‐controlled Walk‐PHaSST study.…”

Section: Discussionsupporting

confidence: 81%

“…As a result of these factors, the prospective clinical investigation of vasodilators approved for precapillary PH (similar to WHO Group 1 PAH) in SCD have largely halted. Despite this classification re‐assignment, many PH centers treating patients with SCD continue to use PAH medications off‐label in those with RHC‐defined precapillary PH, given the limited therapeutic options available 16 . In our clinical experience, some patients have tolerated PDE5‐I long‐term despite warnings against use of this class of medications.…”

Section: Introductionmentioning

confidence: 99%

Long‐term tolerability of phosphodiesterase‐5 inhibitors in pulmonary hypertension of sickle cell disease

Cramer-Bour

¹

,

Ruhl

²

,

Nouraie

³

et al. 2021

European J of Haematology

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Objectives Sickle cell disease‐related pulmonary hypertension (SCD‐PH) is a complex disorder with multifactorial contributory mechanisms. Previous trials have evaluated the efficacy of pulmonary arterial hypertension (PAH) therapies in SCD‐PH with mixed results. We hypothesized that a subset of patients with right heart catheterization (RHC) confirmed disease may benefit from PAH therapy. Methods We performed a retrospective chart review of patients with SCD‐PH diagnosed by RHC who were treated with phosphodiesterase 5 inhibitor (PDE5‐I) therapy for ≥4 months between 2008 and 2019 at two institutions. Results Thirty‐six patients were included in the analysis. The median age (IQR) upon PDE5‐I initiation was 47.5 years (35‐51.5 years); 58% were female and twenty‐nine (81%) had HbSS disease. Of these, 53% of patients had a history of acute chest syndrome, 42% had a history of venous thromboembolism, and 38% had imaging consistent with chronic thromboembolic PH. Patients were treated for a median duration of 25 months (IQR 13‐60 months). Use of PDE5‐I was associated with a significant improvement in symptoms as assessed by NYHA Class (P = .002). Conclusions In SCD patients with PH defined by RHC, PDE5‐I therapy was tolerated long‐term and may improve physical activity.

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“…The final guidelines, including recommendations, were reviewed and approved by all members of the panel. The approach is described in detail in Murad et al 14…”

Section: • Alloimmunizationmentioning

confidence: 99%

American Society of Hematology 2020 guidelines for sickle cell disease: transfusion support

Chou

¹

,

Alsawas

²

,

Fasano

³

et al. 2020

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Background: Red cell transfusions remain a mainstay of therapy for patients with sickle cell disease (SCD), but pose significant clinical challenges. Guidance for specific indications and administration of transfusion, as well as screening, prevention, and management of alloimmunization, delayed hemolytic transfusion reactions (DHTRs), and iron overload may improve outcomes. Objective: Our objective was to develop evidence-based guidelines to support patients, clinicians, and other healthcare professionals in their decisions about transfusion support for SCD and the management of transfusion-related complications. Methods: The American Society of Hematology formed a multidisciplinary panel that was balanced to minimize bias from conflicts of interest and that included a patient representative. The panel prioritized clinical questions and outcomes. The Mayo Clinic Evidence-Based Practice Research Program supported the guideline development process. The Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach was used to form recommendations, which were subject to public comment. Results: The panel developed 10 recommendations focused on red cell antigen typing and matching, indications, and mode of administration (simple vs red cell exchange), as well as screening, prevention, and management of alloimmunization, DHTRs, and iron overload. Conclusions: The majority of panel recommendations were conditional due to the paucity of direct, high-certainty evidence for outcomes of interest. Research priorities were identified, including prospective studies to understand the role of serologic vs genotypic red cell matching, the mechanism of HTRs resulting from specific alloantigens to inform therapy, the role and timing of regular transfusions during pregnancy for women, and the optimal treatment of transfusional iron overload in SCD.

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“…The work of this panel was coordinated with that of 4 other guideline panels (addressing other aspects of SCD) by ASH and the Mayo Clinic Evidence-Based Practice Research Program (funded by ASH under a paid agreement). 14 Project oversight was provided by a coordination panel, which reported to the ASH Guideline Oversight Subcommittee. ASH vetted individuals and appointed them to the guideline panel.…”

Section: Methodsmentioning

confidence: 99%

“…The Mayo Program vetted and retained researchers to conduct systematic reviews of evidence and coordinate the guideline development process, including the use of the GRADE approach. 14 The members of the guideline panel and the Mayo Program team are listed in supplemental File 1.…”

Section: Methodsmentioning

confidence: 99%

Faculty Opinions recommendation of American Society of Hematology 2020 guidelines for sickle cell disease: transfusion support.

Kopko

¹

2020

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature

0

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Background: Red cell transfusions remain a mainstay of therapy for patients with sickle cell disease (SCD), but pose significant clinical challenges. Guidance for specific indications and administration of transfusion, as well as screening, prevention, and management of alloimmunization, delayed hemolytic transfusion reactions (DHTRs), and iron overload may improve outcomes. Objective: Our objective was to develop evidence-based guidelines to support patients, clinicians, and other healthcare professionals in their decisions about transfusion support for SCD and the management of transfusion-related complications. Methods: The American Society of Hematology formed a multidisciplinary panel that was balanced to minimize bias from conflicts of interest and that included a patient representative. The panel prioritized clinical questions and outcomes. The Mayo Clinic Evidence-Based Practice Research Program supported the guideline development process. The Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach was used to form recommendations, which were subject to public comment. Results: The panel developed 10 recommendations focused on red cell antigen typing and matching, indications, and mode of administration (simple vs red cell exchange), as well as screening, prevention, and management of alloimmunization, DHTRs, and iron overload.

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2019 sickle cell disease guidelines by the American Society of Hematology: methodology, challenges, and innovations

Cited by 17 publications

References 18 publications

Long‐term tolerability of phosphodiesterase‐5 inhibitors in pulmonary hypertension of sickle cell disease

Long‐term tolerability of phosphodiesterase‐5 inhibitors in pulmonary hypertension of sickle cell disease

American Society of Hematology 2020 guidelines for sickle cell disease: transfusion support

Faculty Opinions recommendation of American Society of Hematology 2020 guidelines for sickle cell disease: transfusion support.

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