Haploidentical Hematopoietic Stem Cell Transplantation for Patients with Severe Aplastic Anemia—Single-Centre Experience

Round 1

Reviewer 1 Report

Comments and Suggestions for Authors

The text by Vered Stavi et al. describes the outcome of 4 patients undergoing haploidentical transplantation for SAA. The authors report 3 patients who underwent haploidentical transplantation as first line and 1 patient who underwent transplantation due to failure of immunosuppressive therapy. The authors report on 4 young women of non-Caucasian ethnicity who did not have a family donor or MUD, who received a transplant according to the modified Baltimore protocol. The introduction is clear and concise, the results section lacks the timing of the last follow up and the status of chimerism on that date. Authors should report the opinion of the ethics committee or Institutional Review Board regarding the study with the approval number. The authors should specify that this is a pilot study and should be considered as such. The discussion can be greatly reduced by specifying the results of the study compared to those available in the literature with more consolidated approaches.

Comments on the Quality of English Language

English language is fine

Author Response

Thank you very much for your comments. We will appreciate if you will agree to review our manuscript after we did corrections according to your suggestions –

• We added the time of the last follow up visit and the last chimerism to the table.
• We added the ethic committee approval number.
• Thank you for your comment – we emphasized that it is a pilot study.
• The discussion was shortened.

Thank you very much.

Sincerely,

Vered Stavi

Reviewer 2 Report

Comments and Suggestions for Authors

The authors reported 4 cases of SAA treated with haploidentical transplantation. I have some comments.

1. Title  One of four cases is treated with haploidentical peripheral blood stem cell transplantation according to Table 1. “Haploidentical Bone Marrow Transplant for Patients...”　should be amended to “Haploidentical Stem Cell Transplant for Patients...”.

2. Abstract   The authors should describe the abstract of patients. Abstract is not an announcement notice. The authors should include background, case, prognosis, and conclusion, briefly. The abstract must be totally replaced.

3. Methods   At line 70, here are methods, therefore, the authors should not include results. The sentence “ There were three patients ...“　should be erased.

4. Table   The authors should include the severity grade or count of blood cells for better understanding. And the authors should include overall survival time or observation time until now. Just one year of observation (line 128) is not insufficient.

5. Discussion  The authors should discuss umbilical cord blood transplant in addition to MSD BMT.

Author Response

Thank you very much for your comments. We will appreciate if you will agree to review our manuscript after we did corrections according to your suggestions –

1. The title was changed to HAPLOIDENTICAL HEMATOPOIETIC STEM CELL TRANSPLANTION FOR PATIENTS WITH SEVERE APLASTIC ANEMIA –SINGLE CENTRE EXPERIENCE
2. Thank you for your comment. The abstract was replaced.
3. The mentioned line in the method section – “There were three patients “ was removed.
4. We added neut and plt counts prior to transplant. As reticulocytes were tested at different time points we didn’t add it.
5. We added a paragraph about unrelated cord transplants.

Thank you very much.

Sincerely,

Vered Stavi

Reviewer 3 Report

Comments and Suggestions for Authors

This retrospective case report by Stavi et al documented good responses of four young female SAA patients who received HCT from related haploidentical donors. Key references were cited and the report is well-prepared in general. There are, however, a few specific comments/concerns authors may wish to address to further improve this important case report.   

Specific comments:

A key shortfall of this report is the small number of cases. Why only 4 cases from January 2020 to December 2021 were included in the report? New cases in the last two years (January 2022 to December 2023) of the same center should be included, unless a larger scale study is currently ongoing started from 2022 which would prevent the inclusion of new cases.

Data presented in the Table looked goodl! A few key parameters should be added to the Table: a) counts of reticulocytes, neutrophils and platelets immediately prior HCT; b) Most recent chimerism (number of days post HCT).

The statement between lines 209 and 211 “Based on our experience and the emerging literature, we feel a haplo-identical transplant should be offered to young patients with SAA, rather than the conventional immunosuppressive therapy” is very strong with potential clinical implications. Such a strong statement should be associated with reports from major clinical trials containing large numbers of observations, not with a report of only 4 cases. In the current report, case 2 and the other 3 cases all had reasonably good responses. In the original 2020 Blood Advances report, OS was higher in the relapse/refractory cohort than in the treatment-naïve cohort. Thus, based on this reviewer’s interpretation, these observations did not provide any support to the author’s statement, if not providing evidence to the opposite side of the argument.      

Author Response

Thank you very much for your comments. We will appreciate if you will agree to review our manuscript after we did corrections according to your suggestions –

The REB approval is only for the period when there were 4 cases. After these 4 patients, there was a period when there were no haplo-transplants for aplastic anemia. The few done recently have very limited follow-up, and no REB approval. Hence, we would not like to continue with the present study patients.

We added the Neut and plt count prior to transplant. We have not added the reticulocyte counts just before transplant, as the information is not available. We do not do reticulocyte counts on routine blood counts. Hence, the reticulocyte counts near the transplant dates are known, but not just prior to the transplant. Therefore, we feel we should not provide this information as it will not be accurate.

We thank the reviewer for the comment. We have changed the statement for “selected” young patients and softened the statement.

Thank you very much.

Sincerely,

Vered Stavi

Round 2

Reviewer 1 Report

Comments and Suggestions for Authors

Authors fully replid to each questions

Comments on the Quality of English Language

English languages is acceptable

Reviewer 2 Report

Comments and Suggestions for Authors

The authors sufficiently amended the manuscript. I have no further comments.

Reviewer 3 Report

Comments and Suggestions for Authors

The revised manuscript is much improved.