Etuvetidigene Autotemcel for the Treatment of Wiskott–Aldrich Syndrome
Peer-reviewed clinical study; NEJM 2026, 2026 · paper · Clinical trials
Pools data from two clinical studies and an expanded-access program on the gene therapy etu-cel in 27 patients with Wiskott–Aldrich syndrome. Survival at five years was 96%, and severe infections and bleeding events decreased after treatment.
3 findings
Reference or label In the last row of Table 1 the unit is printed as "×10−6 cells/kg" (negative exponent), while the text gives the same dose as "16.9×10^6 per kilogram".
- p. 4 Table 1, last row
Median no. of CD34+ cells infused (range) — ×10−6
- p. 4 Results, Participants
16.9×106 per kilogram
- p. 4 Table 1
Median no. of CD34+ cells infused (range) — ×10−6 cells/kg
- p. 4 Table 1, last row
×10−6 cells/kg
Judge's note: The p4 crop shows a superscript −6 in the Table 1 row label. The text in the right column of p4 shows a superscript 6. This is a unit or label error in the table; the data are fine.
Second check: another model agreed
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Reference or label The Fig. 3C y-axis label reads "Median Count (10−9/liter)" with a negative exponent, while the text gives platelet counts in units of ×10^9 per liter.
- p. 8 Figure 3C y-axis
Median Count (10–9/liter)
- p. 9 Platelets text
18.0×109 per liter
Judge's note: The p8 crop shows a superscript −9 in the axis label. The p9 text and the p10 Discussion use ×10^9 per liter. The axis unit is mislabelled.
Second check: another model agreed
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Reference or label Reference 4 lists a spurious fourth author taken from the title: "Vallée TC, Albert MH, Pai S-Y, How I. How I treat Wiskott-Aldrich syndrome. Blood 2025;146:41-51."
- p. 13 Reference 4
Vallée TC, Albert MH, Pai S-Y, How I. How I treat Wiskott-Aldrich syndrome.
Judge's note: The p. 13 reference-list image confirms the entry as quoted. "How I." repeats the opening words of the title "How I treat…", which is the usual pattern of a reference-manager parsing error. The fix is to remove "How I." from the author list. The data are unaffected; only the citation entry is wrong.
Second check: another model agreed
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8 minor (typos and the like; most checked by the judge only)
Reference or label The Discussion sentence "This finding is in line with those from other studies that show a correlation between platelet counts and gene marking in myeloid cells and bone marrow." carries no reference.
- p. 10 Discussion, paragraph 2
This finding is in line with those from other studies that show a correlation between platelet counts and gene marking in myeloid cells and bone marrow.
Only the authors can say what is right here.
Judge's note: The p10 image shows no superscript at the end of this sentence, while the neighbouring external claims carry citations (18, 24, 25, 26, 27). Only the authors can say which studies are meant.
Second check: another model agreed
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Wording The text says the platelet increase "was sustained at all available later time points up to year 13 (Fig. 3C)", but the x-axis of Fig. 3C ends at year 12 (n=2).
- p. 9 Results, Platelets
the increase compared with baseline was sustained at all available later time points up to year 13 (Fig. 3C)
- p. 8 Figure 3C x-axis
Years since Gene Therapy
- p. 9 Platelets, first paragraph
sustained at all available later time points up to year 13 (Fig. 3C)
- p. 8 Fig. 3C x-axis
12
Only the authors can say what is right here.
Judge's note: The Fig. 3C crop has its last plotted point at year 12, with 2 participants. The legend says "Results based on data from fewer than two participants were omitted", which could explain why a year-13 value is missing, but the cited figure does not show the year-13 claim. Only the authors can say whether a single-participant year-13 value exists and where it is reported.
Second check: another model agreed
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Typo The Acknowledgments thank "the Children’s Hospital of Atlanta", but the Methods, the figure legend and affiliation 6 name the site "Children’s Healthcare of Atlanta".
- p. 12 Acknowledgments
and the Children’s Hospital of Atlanta for their professional care
- p. 2 Methods
Children’s Health-care of Atlanta (1 participant)
- p. 12 Acknowledgments
the Children’s Hospital of Atlanta for their professional care
- p. 12 Acknowledgments
the Children’s Hospital of Atlanta
Judge's note: The p12 crop shows "Children’s Hospital of Atlanta". The p2 Methods ("Children’s Healthcare of Atlanta (1 participant)"), the figure legend and affiliation 6 all use "Healthcare". The acknowledgment refers to the same single site, so the name is inconsistent within the paper.
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Typo Reference 16 ("eczema score,16", End Points) is cited before references 14 and 15, which are first cited later in Statistical Analysis, so the citation-order numbering is broken.
- p. 3 End Points
Eczema was classified according to the eczema score,16
- p. 3 Statistical Analysis
calculated with the complementary log–log transforma-tion.14
Judge's note: On the p3 image, End Points (end of the left column and top of the right column) cites ref 16, and Statistical Analysis below it then cites 14 and 15. I found no earlier citation of 14 or 15. The reference list shows 14 Lawless, 15 Garwood and 16 Imai. Each citation's content looks appropriate, so the only problem is the numbering order.
Second check: none (judge only)
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Typo Table 1 footnote ‡ ends "rather than at baseline" with no closing period; the other footnotes end with one.
- p. 4 Table 1, footnote ‡
blood stem-cell collection (representing the start of treatment phase), rather than at baseline
- p. 4 Table 1 footnote ‡
rather than at baseline
Judge's note: The p4 crop confirms there is no period after "baseline", while footnotes *, †, § and ¶ all end with periods.
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Typo The Figure 3 legend spells "Henoch–Schonlein purpura" without the umlaut (Henoch–Schönlein).
- p. 9 Figure 3 legend
Henoch–Schonlein purpura
Judge's note: The p9 crop shows "Schonlein" in the rendered text, so this is not an extraction artefact. The spelling of the eponym is general knowledge.
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Typo Table 2 footnote † writes "(e.g. blood tests)" without a comma and "(e.g., imaging studies)" with one, in the same sentence.
- p. 11 Table 2, footnote †
(e.g. blood tests)
Judge's note: The upright p11 image confirms both forms in footnote †. Elsewhere the paper uses "e.g.," (for example, p6 "(e.g., sple-nectomy)").
Second check: none (judge only)
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Typo The surname is spelled "Loïc Dupre" (no accent) in the Acknowledgments (p. 12) but "Dupré L" in Reference 2 (p. 13).
- p. 12 Acknowledgments
Loïc Dupre and Samantha Scaramuzza
- p. 13 Reference 2
Dupré L, Castanon I, Boztug K.
- p. 12 Acknowledgments
Loïc Dupre
- p. 13 Reference 2
Dupré L, Castanon I
Only the authors can say what is right here.
Judge's note: Rests on outside knowledge: that the person acknowledged and the first author of reference 2 are the same person, whose name is spelled Dupré. The page images confirm both spellings: the Acknowledgments on p. 12 read "Loïc Dupre and Samantha Scaramuzza", and Reference 2 on p. 13 reads "Dupré L, Castanon I, Boztug K." The first name keeps its diaeresis, so the missing accent on the surname looks like an omission. This is a cosmetic inconsistency.
Second check: none (judge only)