Enhancing the Management of Pediatric Sickle Cell Disease by Integrating Functional Evaluation to Mitigate the Burden of Vaso-Occlusive Crises

Round 1

Reviewer 1 Report

Comments and Suggestions for Authors

Review of Enhancing Sickle Cell Management by Integrating Functional Evaluation to Mitigate the Burden of Vaso-Occlusive Crisis

 

1/9/2024

 

This is an opinion piece about how multiple rehabilitation services could improve pediatric sickle cell pain and stroke care. The authors have a good grasp of the needed care of sickle cell patient and of how rehabilitation techniques and technology may help. Rehabilitation services are sorely needed, under utilized, and underfunded. The message of this opinion piece, and the research behind it, are important and need to be more widely discussed.

I would re-consider after revision.

Main Points:

1.       While agreeing with most of the author’s points, it was hard to see how this would be implemented in detail in clinics, much less under resourced clinics. Many of the rehab techniques and technology have a hopeful, but unproven, effect on the pathophysiology of sickle cell disease. They would likely improve quality of life after the damage of a sickle cell complication.  I kept trying to visualize a chart with the problems, the functional assessments, and the expected outcomes (with citations).

2.       My take-away what that there is great hope in using these newer technologies to find where earlier interventions may help, and in using them to monitor improvements in the functional assessments.  A paragraph outlining a specific research agenda would be welcomed.

3.       The authors are quite correct that focusing on function is critical to quality of life regardless of available resources. Formalizing functional assessments could change care for many patients.

Minor Points:

Title:

The paper is focused on children, so the world pediatrics should appear in the title to better characterize the paper.

Abstract:  

The abstract was well written. The point about lack of rehab services, especially neuro rehab is spot on. They promised a focus on VOC and strokes. Little was said about strokes, and stroke was not in the concluding paragraph.

Keywords: LMIC was not defined at this point

Introduction: 

Line 61 : …, resembling cognitive defects,   maybe better “reflecting” or “associated with”

Lines 86 – 90: The author cites his own paper in support of the premise of this paper. Adding other citations would strengthen his argument.

Lines 92 – 94: The line wasn’t clear in meaning. I was left with the impression that after a stroke, functional evaluation may lead to less VOC.  This doesn’t establish a causal link for that conclusion.

Epidemiology of Vaso-Occlusive Crisis in SCD patent

This was an interesting section. The authors approach, supported buy a retrospective chart review,  to categorizing sickle cell pain was a strength in LMIC settings. A line or two comparing it to other studies on sickle cell pain categorizations could strengthen the paper’s argument that his functional classification would better direct therapies.

Line 102: Figure 1 presents the complications of sickle cell disease. I think the author is trying to say that all complications come from vascular occlusions.  The most common usage of VOC implies pain, but much end organ damage happens without acute pain.  Though I must mention that several studies of sickle cell lump many complications under sickle cell crisis.  Perhaps “The most common VOC associated complications are presented in Figure 1.”

Lines 124 -125: It would be more clear to state: …, moderate when the score was 4 or 5, and…”

Functional Evaluation as a More Multidimensional Diagnostic Tool

This section could have been improved by citing examples of the studies that have used monitoring for diagnostics, then expanding on how the functional rehab approach could expand on their usefulness.

The Role of Functional Evaluation in Improving Vaso-Occlusive Crisis Management

Lines 183 to 187: Finding early enough signals of pending crises to allow treatment is the holy grail of sickle cell pain management.  This is the strongest point in the paper. However,  stroke prevention isn’t likely to have a clear prodrome. Picking up a stroke as early as possible could arguably lead to quicker transfusions and better outcomes.

Challenges and Opportunities

Line 222: I think the WHO report addressed all rehab services. Were there any sickle cell specific recommendations? If so, they should be highlighted.

Dr. Elliott Vichinsky often has said that if sickle cell didn’t have pain as a component, most sufferers would be in neurorehabilitation facilities. Much evolving data shows the early and progression cognitive challenges of sickle cell disease, where rehab is greatly needed.

Lines 248 to 250 were overstated about how rehabilitation would help in stroke prevention, which is a different pathophysiology than pain. Patients with lower average hemoglobin have less pain, but more strokes. The authors could state how that connection might work, or state that comprehensive approach, besides the critical post-stroke rehab, could possibly  contribute to secondary stroke  prevention.

Conclusion

The “In conclusion” paragraph does not mention stroke (that was highlighted in the abstract as a focus). Would be consistent.  

Author Response

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Author Response File: Author Response.pdf

Reviewer 2 Report

Comments and Suggestions for Authors

The manuscript ”Enhancing Sickle Cell Disease Management by Integrating Functional Evaluation to Mitigate the Burden of Vaso-Occlusive Crises” by Boma et al., is a short article emphasizing on the management strategies in pediatric patients of Sickle Cell disease (SCD) having Vaso-Occlusive crisis (VOC). Authors have discussed the role of rehabilitation services in pediatric patients especially with stroke and VOC, can improve the overall outcome and mitigate the challenges faced by patients and their families.

In general, the study has a novel rationale of combining the medicines with rehabilitation strategies to mitigate the pain and thus VOC and stroke in pediatric patients, the study does not elaborate on the strategies or the technologies that could be used in SCD and their accessibility to the patients. The article cannot be accepted in the present form. The quality of the manuscript could be improvised by addressing a few issues:

General: Authors have not explained in details the rehabilitation strategies that are available in general and that are available in Lubumbashi. In addition, the authors suggested that by combining rehabilitation services integrating with technology- supported therapy would prevent stroke, I believe the authors meant to improve the condition of stroke patients but not prevention. If not, authors must explain.

P1, line 45: Authors could cite Lancet 2023, to address the global burden of SCD (PMID:37331373).

P3, line 112: ‘to’ in the sentence “To evaluate the magnitude of this problem and to determine which type of crisis was to most prevalent” can be deleted.

P3, line 115: Authors must add the abbreviation of CRDL.

P4, line 130: Authors must add a note if the VOC severity was based only on pain assessment or hospitalization, as VOC often end with hospitalization. Perhaps authors can also add a note if these patients underwent rehabilitation services.

Author Response

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Author Response File: Author Response.pdf

Reviewer 3 Report

Comments and Suggestions for Authors

The concept described is of interest to all who manage SCD patients and it is a minority that actually consider rehabilitation in  the management of VOC. Even fewer if any have used technology assisted functional assessment. This makes this paper of great interest to such treatment units recognising the need for a multidisciplinary and comprehensive approach. What I find not convincing in this presentation is the introduction of  such services in resource limited settings. The authors have made a good case concerning the usefulness of rehabilitation but have not argued how cost-effectiveness is achieved  in a poor country with 40000 affected newborn added each year,  and limited skilled personnel. The expense and availability of suitable sensors, teaching each patient the usage in a home environment, the need data processing, makes the adoption by countries with limited resources problematic and so more argument is needed on how resources can be saved, how patients can be reached in a country where basic needs such as hydroxyurea and blood foe transfusion are scarce. In addition a patient cohort of 838 patients is mentioned but what devises were used and whether there was demonstrable benefit to these patients is not mentioned yet to those willing to adopt the techniques these are the questions that must be answered. The authors recognise the 'notable obstacles' but the generalisation that there is need to enhance efforts to make available skilled personnel and secure funding are discussed in one sentence. In conclusion this paper recommends a new approach for limited resource countries without further discussion on say advocacy with authorities based on evidence that funding would enhance outcomes for patients. Is integrating technology -supported therapy worth supporting for SCD? this cannot be simply based on theory evidence is required. 

Comments on the Quality of English Language

English is generally very good. Minor points in lines 122-123 the sentence for children... seems incomplete. 

Sentence 188-189 needs rephrasing. 

Author Response

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Author Response File: Author Response.pdf

Round 2

Reviewer 2 Report

Comments and Suggestions for Authors

The manuscript entitled "Enhancing Pediatrics Sickle Cell Disease Management by Integrating Functional Evaluation to Mitigate the Burden of Vaso-Occlusive Crises" by Boma et al., is well written. The paper highlights the need for technology-assisted strategies to alleviate neuropathic problems and also their availability in low- and middle-income countries. SCD management is a challenge in these countries due to unavailability of the resources. The authors have addressed all the reviewer comments satisfactorily.

Reviewer 3 Report

Comments and Suggestions for Authors

My concerns about providing rehabilitation and mobile sensors in a low income setting have been answered especially in page 7 of the new manuscript. This point was fully discussed in the original version. The paper is now ready for publication